Brian K. Kaspar, Ph.D. :: Nationwide Children's Hospital, Columbus, Ohio

Brian K. Kaspar, PhD

Brian K. Kaspar, PhD

Center for Gene Therapy
Principal Investigator

Principal Investigator

Neurology Residency

Neuromuscular Disorders
Principal Investigator

Principal Investigator

Contact Information

The Research Institute at Nationwide Children's Hospital
700 Childrens Drive
Columbus, OH 43205 [ map ]
PH: (614) 722-2700
FX: (614) 355-5247
Email Me


Brian K. Kaspar, PhD is a principal investigator in the Center for Gene Therapy at The Research Institute at Nationwide Children’s Hospital and Associate Professor in the Department of Pediatrics and Department of Neuroscience at The Ohio State University College of Medicine. In 2013, Dr. Kaspar was named Fellow of the American Association for the Advancement of Science (AAAS).


  • Male

Languages Spoken:

  • English

Research Interests

Research Center:

Areas of Interest:

  • The Kaspar Laboratory focuses on basic and translational studies related to neurological and neuromuscular disorders. The laboratory has strengths in animal models of neurodegenerative and neuromuscular disease, gene delivery, and stem cell biology. A main focus of the Kaspar laboratory is centered on the mechanism(s) of neurodegeneration in Amyotrophic Lateral Sclerosis (ALS). We employ rodent models of this disease to investigate various cell type involvements in disease onset and progression. Furthermore, we are actively developing novel methods to deliver genes and therapies more efficiently to the nervous system and testing novel targets to combat this debilitating, lethal disease. The laboratory also investigates the biological control of embryonic and adult derived stem cells. Our current studies with stem cells are evaluating cell cycle regulation along with developing methods for intricate control of differentiation to defined cellular phenotypes, such as complex motor neurons. Finally, our laboratory works on muscle enhancing strategies in order to combat musculoskeletal disorders. We have identified that follistatin is a potent antagonist of myostatin, and when delivered by a one-time gene delivery to skeletal muscle, enhances muscle size and mass. Interestingly, our work has demonstrated that follistatin reduces the inflammatory environment in dystrophic muscles and induces improvements in functional strength in rodent models of Duchene Muscular Dystrophy. We are currently involved with developing a human clinical trial for Inclusion Body Myositis with Dr. Jerry Mendell.

Education and Training

Post Doctoral

  • University of California, San Diego
    Date Completed: 06/30/2003

Post Doctoral

  • Salk Institute for Biological Studies
    Date Completed: 06/30/2004


  • Weber-Adrian,D; Thevenot,E; O'Reilly,M,A; Oakden,W; Akens,M,K; Ellens,N; Markham-Coultes,K; Burgess,A; Finkelstein,J; Yee,A,JM; Whyne,C,M; Foust,K,D; Kaspar,B,K; Stanisz,G,J; Chopra,R; Hynynen,K; Aubert,I. 2015. Gene delivery to the spinal cord using MRI-guided focused ultrasound.  GENE THERAPY. Vol. 22, no. 7. (July): 568-577.
  • Israelson,Adrian; Ditsworth,Dara; Sun,Shuying; Song,SungWon; Liang,Jason; Hruska-Plochan,Marian; McAlonis-Downes,Melissa; Abu-Hamad,Salah; Zoltsman,Guy; Shani,Tom; Maldonado,Marcus; Bui,Anh; Navarro,Michael; Zhou,Huilin; Marsala,Martin; Kaspar,Brian,K; Da Cruz,Sandrine; Cleveland,Don,W. 2015. Macrophage Migration Inhibitory Factor as a Chaperone Inhibiting Accumulation of Misfolded SOD1.  NEURON. Vol. 86, no. 1. (April): 218-232.
  • Israelson,Adrian; Ditsworth,Dara; Sun,Shuying; Song,SungWon; Liang,Jason; Hruska-Plochan,Marian; McAlonis-Downes,Melissa; Abu-Hamad,Salah; Zoltsman,Guy; Shani,Tom; Maldonado,Marcus; Bui,Anh; Navarro,Michael; Zhou,Huilin; Marsala,Martin; Kaspar,Brian,K; Da Cruz,Sandrine; Cleveland,Don,W. 2015. Macrophage Migration Inhibitory Factor as a Chaperone Inhibiting Accumulation of Misfolded SOD1.  Neuron. Vol. 86, no. 1. (April): 218-232.
  • Cheever,Thomas,R; Berkley,Dale; Braun,Serge; Brown,Robert,H; Byrne,Barry,J; Chamberlain,Jeffrey,S; Cwik,Valerie; Duan,Dongsheng; Federoff,Howard,J; High,Katherine,A; Kaspar,Brian,K; Klinger,Katherine,W; Larkindale,Jane; Lincecum,John; Mavilio,Fulvio; McDonald,Cheryl,L; McLaughlin,James; McLeod,Bonnie,Weiss; Mendell,Jerry,R; Nuckolls,Glen; Stedman,Hansell,H; Tagle,Danilo,A; Vandenberghe,Luk,H; Wang,Hao; Wernett,Pamela,J; Wilson,James,M; Porter,John,D; Gubitz,Amelie,K. 2015. Perspectives on Best Practices for Gene Therapy Programs.  HUMAN GENE THERAPY. Vol. 26, no. 3. (March): 127-133.
  • Meyer,Kathrin; Ferraiuolo,Laura; Schmelzer,Leah; Braun,Lyndsey; McGovern,Vicki; Likhite,Shibi; Michels,Olivia; Govoni,Alessandra; Fitzgerald,Julie; Morales,Pablo; Foust,Kevin,D; Mendell,Jerry,R; Burghes,Arthur,HM; Kaspar,Brian,K. 2015. Improving Single Injection CSF Delivery of AAV9-mediated Gene Therapy for SMA: A Dose-response Study in Mice and Nonhuman Primates.  MOLECULAR THERAPY. Vol. 23, no. 3. (March): 477-487.
  • Duque,Sandra,I; Arnold,W,David; Odermatt,Philipp; Li,Xiaohui; Porensky,Paul,N; Schmelzer,Leah; Meyer,Kathrin; Kolb,Stephen,J; Schuemperli,Daniel; Kaspar,Brian,K; Burghes,Arthur,HM. 2015. A Large Animal Model of Spinal Muscular Atrophy and Correction of Phenotype.  ANNALS OF NEUROLOGY. Vol. 77, no. 3. (March): 399-414.
  • Mendonca,Liliana,S; Nobrega,Clevio; Hirai,Hirokazu; Kaspar,Brian,K; de Almeida,Luis,Pereira. 2015. Transplantation of cerebellar neural stem cells improves motor coordination and neuropathology in Machado-Joseph disease mice.  BRAIN. Vol. 138, no. February: 320-335.
  • Hesp,Zoe,C; Goldstein,Evan,A; Miranda,Carlos,J; Kaspar,Brain,K; McTigue,Dana,M. 2015. Chronic Oligodendrogenesis and Remyelination after Spinal Cord Injury in Mice and Rats.  JOURNAL OF NEUROSCIENCE. Vol. 35, no. 3. (January): 1274-1290.
  • Mendell,Jerry,R; Sahenk,Zarife; Malik,Vinod; Gomez,Ana,M; Flanigan,Kevin,M; Lowes,Linda,P; Alfano,Lindsay,N; Berry,Katherine; Meadows,Eric; Lewis,Sarah; Braun,Lyndsey; Shontz,Kim; Rouhana,Maria; Clark,Kelly,Reed; Rosales,Xiomara,Q; Al-Zaidy,Samiah; Govoni,Alessandra; Rodino-Klapac,Louise,R; Hogan,Mark,J; Kaspar,Brian,K. 2015. A Phase 1/2a Follistatin Gene Therapy Trial for Becker Muscular Dystrophy.  MOLECULAR THERAPY. Vol. 23, no. 1. (January): 192-201.
  • Thomsen,Gretchen,M; Gowing,Genevieve; Latter,Jessica; Chen,Maximus; Vit,Jean-Philippe; Staggenborg,Kevin; Avalos,Pablo; Alkaslasi,Mor; Ferraiuolo,Laura; Likhite,Shibi; Kaspar,Brian,K; Svendsen,Clive,N. 2014. Delayed Disease Onset and Extended Survival in the SOD1(G93A) Rat Model of Amyotrophic Lateral Sclerosis after Suppression of Mutant SOD1 in the Motor Cortex.  JOURNAL OF NEUROSCIENCE. Vol. 34, no. 47. (November): 15587-15600.
  • Lampe,Sara,EGombash; Kaspar,Brian,K; Foust,Kevin,D. 2014. Intravenous Injections in Neonatal Mice.  JOVE-JOURNAL OF VISUALIZED EXPERIMENTS. Vol. ., no. 93. (November): ee52037.
  • Meyer,Kathrin; Kaspar,Brian,K. 2014. Making Sense of Pain: Are Pluripotent Stem Cell-derived Sensory Neurons a New Tool for Studying Pain Mechanisms?.  MOLECULAR THERAPY. Vol. 22, no. 8. (August): 1403-1405.
  • Sahenk,Zarife; Galloway,Gloria; Clark,Kelly,Reed; Malik,Vinod; Rodino-Klapac,Louise,R; Kaspar,Brian,K; Chen,Lei; Braganza,Cilwyn; Montgomery,Chrystal; Mendell,Jerry,R. 2014. AAV1.NT-3 Gene Therapy for Charcot-Marie-Tooth Neuropathy.  MOLECULAR THERAPY. Vol. 22, no. 3. (March): 511-521.
  • Frakes,Ashley,E; Ferraiuolo,Laura; Haidet-Phillips,Amanda,M; Schmelzer,Leah; Braun,Lyndsey; Miranda,Carlos,J; Ladner,Katherine,J; Bevan,Adam,K; Foust,Kevin,D; Godbout,Jonathan,P; Popovich,Phillip,G; Guttridge,Denis,C; Kaspar,Brian,K. 2014. Microglia Induce Motor Neuron Death via the Classical NF-kappa B Pathway in Amyotrophic Lateral Sclerosis.  NEURON. Vol. 81, no. 5. (March): 1009-1023.
  • Camboni,Marybeth; Wang,Chiou-Miin; Miranda,Carlos; Yoon,Jung,Hae; Xu,Rui; Zygmunt,Deborah; Kaspar,Brian,K; Martin,Paul,T. 2014. Active and passive immunization strategies based on the SDPM1 peptide demonstrate pre-clinical efficacy in the APPswePSEN1dE9 mouse model for Alzheimer's disease.  Neurobiology of disease. Vol. 62, no. February: 31-43.
  • Meyer,Kathrin; Ferraiuolo,Laura; Miranda,Carlos,J; Likhite,Shibi; McElroy,Sohyun; Renusch,Samantha; Ditsworth,Dara; Lagier-Tourenne,Clotilde; Smith,Richard,A; Ravits,John; Burghes,Arthur,H; Shaw,Pamela,J; Cleveland,Don,W; Kolb,Stephen,J; Kaspar,Brian,K. 2014. Direct conversion of patient fibroblasts demonstrates non-cell autonomous toxicity of astrocytes to motor neurons in familial and sporadic ALS.  PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA. Vol. 111, no. 2. (January): 829-832.
  • Gombash Lampe,Sara,E; Kaspar,Brian,K; Foust,Kevin,D. 2014. Intravenous injections in neonatal mice.  Journal of visualized experiments : JoVE. Vol. ., no. 93. (January): ee52037.
  • Bevan, Kaspar. 2014. CNS Gene Therapy Utilizing Intravenously Administered Viral Vectors. In Drug Devliery to the Brain. Edited by Crommelin and Lipper. New York: Springer.
  • Arnold, Porensky, McGovern, Iyer, Duque, Li, Meyer, Schmelzer, Kaspar, Kolb, Kissel, Burghes. 2014. Electrophysiological Biomarkers in Spinal Muscular Atrophy: Preclinical Proof of Concept.  Annals in Clinical Translational Neurology. Vol. 1, no. 1. (January): e34.
  • Rodino-Klapac,Louise,R; Janssen,Paul,ML; Shontz,Kimberly,M; Canan,Benjamin; Montgomery,Chrystal,L; Griffin,Danielle; Heller,Kristin; Schmelzer,Leah; Handy,Chalonda; Clark,K,Reed; Sahenk,Zarife; Mendell,Jerry,R; Kaspar,Brian,K. 2013. Micro-dystrophin and follistatin co-delivery restores muscle function in aged DMD model.  HUMAN MOLECULAR GENETICS. Vol. 22, no. 24. (December): 4929-4937.
  • Foust,Kevin,D; Salazar,Desiree,L; Likhite,Shibi; Ferraiuolos,Laura; Ditsworth,Dara; Ilieva,Hristelina; Meyer,Kathrin; Schmelzer,Leah; Braun,Lyndsey; Cleveland,Don,W; Kaspar,Brian,K. 2013. Therapeutic AAV9-mediated Suppression of Mutant SOD1 Slows Disease Progression and Extends Survival in Models of Inherited ALS.  MOLECULAR THERAPY. Vol. 21, no. 12. (December): 2148-2159.
  • Foust,Kevin,D; Salazar,Desirée,L; Likhite,Shibi; Ferraiuolo,Laura; Ditsworth,Dara; Ilieva,Hristelina; Meyer,Kathrin; Schmelzer,Leah; Braun,Lyndsey; Cleveland,Don,W; Kaspar,Brian,K. 2013. Therapeutic AAV9-mediated suppression of mutant SOD1 slows disease progression and extends survival in models of inherited ALS.  Molecular therapy : the journal of the American Society of Gene Therapy. Vol. 21, no. 12. (December): 2148-2159.
  • Kaspar,Kai. 2013. A Weighty Matter: Heaviness Influences the Evaluation of Disease Severity, Drug Effectiveness, and Side Effects.  PLOS ONE. Vol. 8, no. 11. (November): ee78307.
  • Kaspar,Kai; Krull,Janna. 2013. Incidental Haptic Stimulation in the Context of Flirt Behavior.  JOURNAL OF NONVERBAL BEHAVIOR. Vol. 37, no. 3. (September): 165-173.
  • Garg,Saurabh,K; Lioy,Daniel,T; Cheval,Helene; McGann,James,C; Bissonnette,John,M; Murtha,Matthew,J; Foust,Kevin,D; Kaspar,Brian,K; Bird,Adrian; Mandel,Gail. 2013. Systemic Delivery of MeCP2 Rescues Behavioral and Cellular Deficits in Female Mouse Models of Rett Syndrome.  JOURNAL OF NEUROSCIENCE. Vol. 33, no. 34. (August): 13612-13620.
  • Ferraiuolo, Meyer, Kaspar. 2013. Chapter 6: The use of Human Samples to Study Familial and Sporadic Amyotrophic Lateral Sclerosis: New Frontiers and Challenges. In Current Advances in Amyotrophic Lateral Sclerosis. New York: InTech.
  • Ferraiuolo,Laura; Frakes,Ashley; Kaspar,Brian,K. 2013. Neural Stem Cells as a Therapeutic Approach for Amyotrophic Lateral Sclerosis.  MOLECULAR THERAPY. Vol. 21, no. 3. (March): 503-505.
  • Meyer,Kathrin; Miranda,Carlos,J; Kaspar,Brian,K. 2013. Transplantation of Gene-Corrected Motor Neurons as a Therapeutic Strategy for Spinal Muscular Atrophy.  MOLECULAR THERAPY. Vol. 21, no. 3. (March): 502-503.
  • Kaspar,Kai. 2013. Washing One's Hands After Failure Enhances Optimism but Hampers Future Performance.  SOCIAL PSYCHOLOGICAL AND PERSONALITY SCIENCE. Vol. 4, no. 1. (January): 69-73.
  • Burghes,Arthur; Porensky,Paul; Mitrpant,Charlermchai; McGovern,Vicki; Bevan,Adam; Foust,Kevin; Kaspar,Brian; Wilton,Stephen. 2012. Treatment of Spinal Muscular Atrophy with Antisense Morpholinos that Alter Splicing of SMN2 Good Distribution in Neonates and Adult Animals.  NUCLEIC ACID THERAPEUTICS. Vol. 22, no. 6. (December): A10-A11.
  • Thevenot,Emmanuel; Jordao,Jessica,F; O'Reilly,Meaghan,A; Markham,Kelly; Weng,Ying-Qi; Foust,Kevin,D; Kaspar,Brian,K; Hynynen,Kullervo; Aubert,Isabelle. 2012. Targeted Delivery of Self-Complementary Adeno-Associated Virus Serotype 9 to the Brain, Using Magnetic Resonance Imaging-Guided Focused Ultrasound.  HUMAN GENE THERAPY. Vol. 23, no. 11. (November): 1144-1155.
  • Mendell,Jerry,R; Rodino-Klapac,Louise; Sahenk,Zarife; Malik,Vinod; Kaspar,Brian,K; Walker,Christopher,M; Clark,K,Reed. 2012. Gene therapy for muscular dystrophy: Lessons learned and path forward.  NEUROSCIENCE LETTERS. Vol. 527, no. 2. (October): 90-99.
  • Ferraiuolo,Laura; Kaspar,Brian,K. 2012. Gene Delivery Improvement for Treating the Lysosomal Storage Disorder Metachromatic Leukodystrophy.  HUMAN GENE THERAPY. Vol. 23, no. 8. (August): 793-795.
  • Miranda,Carlos,J; Braun,Lyndsey; Jiang,Yuying; Hester,Mark,E; Zhang,Ling; Riolo,Matthew; Wang,Haijuan; Rao,Meghan; Altura,Rachel,A; Kaspar,Brian,K. 2012. Aging brain microenvironment decreases hippocampal neurogenesis through Wnt-mediated survivin signaling.  AGING CELL. Vol. 11, no. 3. (June): 542-552.
  • Kota,Janaiah; Shilling,Christopher; Montgomery,Chrystal; Lewis,Sarah; Bevan,Adam; Shontz,Kim; Kaminoh,Yuuki; Heller,Kristin; Rosales,Xiomara; Viollet,Laurence; Flanigan,Kevin; Clark,K,Reed; Kaspar,Brian; Sahenk,Zarife; Mendell,Jerry. 2012. Adeno Associated Virus (AAV) Mediated Follistatin Gene Transfer Toxicology Studies in Preparation of Phase I/II Clinical Trial.  MOLECULAR THERAPY. Vol. 20, no. May: S72-S72.
  • Samaranch,Lluis; Salegio,Ernesto,A; San Sebastian,Waldy; Kells,Adrian,P; Foust,Kevin,D; Bringas,John,R; Lamarre,Clementine; Forsayeth,John; Kaspar,Brian,K; Bankiewicz,Krystof,S. 2012. Adeno-Associated Virus Serotype 9 Transduction in the Central Nervous System of Nonhuman Primates.  HUMAN GENE THERAPY. Vol. 23, no. 4. (April): 382-389.
  • Porensky,Paul,N; Mitrpant,Chalermchai; McGovern,Vicki,L; Bevan,Adam,K; Foust,Kevin,D; Kaspar,Brain,K; Wilton,Stephen,D; Burghes,Arthur,HM. 2012. A single administration of morpholino antisense oligomer rescues spinal muscular atrophy in mouse.  HUMAN MOLECULAR GENETICS. Vol. 21, no. 7. (April): 1625-1638.
  • Lowes,Linda,Pax; Alfano,Lindsay; Viollet,Laurence; Rosales,Xiomara,Quintero; Sahenk,Zarife; Kaspar,Brian,K; Clark,K,Reed; Flanigan,Kevin,M; Mendell,Jerry,R; McDermott,Michael,P. 2012. Knee extensor strength exhibits potential to predict function in sporadic inclusion-body myositis.  MUSCLE & NERVE. Vol. 45, no. 2. (February): 163-168.
  • Fidler,Jonathan,A; Treleaven,Christopher,M; Frakes,Ashley; Tamsett,Thomas,J; McCrate,Mary; Cheng,Seng,H; Shihabuddin,Lamya,S; Kaspar,Brian,K; Dodge,James,C. 2011. Disease progression in a mouse model of amyotrophic lateral sclerosis: the influence of chronic stress and corticosterone.  FASEB JOURNAL. Vol. 25, no. 12. (December): 4369-4377.
  • Beastrom,Nicholas; Lu,Haiyan; Macke,Allison; Canan,Benjamin,D; Johnson,Eric,K; Penton,Christopher,M; Kaspar,Brian,K; Rodino-Klapac,Louise,R; Zhou,Lan; Janssen,Paul,ML; Montanaro,Federica. 2011. mdx(5cv) Mice Manifest More Severe Muscle Dysfunction and Diaphragm Force Deficits than Do mdx Mice.  AMERICAN JOURNAL OF PATHOLOGY. Vol. 179, no. 5. (November): 2464-2474.
  • Bevan,Adam,K; Duque,Sandra; Foust,Kevin,D; Morales,Pablo,R; Braun,Lyndsey; Schmelzer,Leah; Chan,Curtis,M; McCrate,Mary; Chicoine,Louis,G; Coley,Brian,D; Porensky,Paul,N; Kolb,Stephen,J; Mendell,Jerry,R; Burghes,Arthur,HM; Kaspar,Brian,K. 2011. Systemic Gene Delivery in Large Species for Targeting Spinal Cord, Brain, and Peripheral Tissues for Pediatric Disorders.  MOLECULAR THERAPY. Vol. 19, no. 11. (November): 1971-1980.
  • White,Robin,E; Rao,Meghan; Gensel,John,C; McTigue,Dana,M; Kaspar,Brian,K; Jakeman,Lyn,B. 2011. Transforming Growth Factor alpha Transforms Astrocytes to a Growth-Supportive Phenotype after Spinal Cord Injury.  JOURNAL OF NEUROSCIENCE. Vol. 31, no. 42. (October): 15173-15187.
  • Hester,Mark,E; Murtha,Matthew,J; Song,SungWon; Rao,Meghan; Miranda,Carlos,J; Meyer,Kathrin; Tian,Jinbin; Boulting,Gabriella; Schaffer,David,V; Zhu,Michael,X; Pfaff,Samuel,L; Gage,Fred,H; Kaspar,Brian,K. 2011. Rapid and Efficient Generation of Functional Motor Neurons From Human Pluripotent Stem Cells Using Gene Delivered Transcription Factor Codes.  MOLECULAR THERAPY. Vol. 19, no. 10. (October): 1905-1912.
  • Burghes,Arthur; Foust,Kevin; McGovern,Vicki; Poresnsky,Paul; Bevan,Adam; Duque,Sandra; Le,Thanh; Iyer,Chitra; Laporte,Aurelie; Alwine,Isaac; Mitrpant,Chalermchai; Wilton,Steve; Kaspar,Brian. 2011. Development and preclinical evaluation of therapies for spinal muscular atrophy in mice.  TRANSGENIC RESEARCH. Vol. 20, no. 5. (October): 1143-1144.
  • Haidet-Phillips,Amanda,M; Hester,Mark,E; Miranda,Carlos,J; Meyer,Kathrin; Braun,Lyndsey; Frakes,Ashley; Song,SungWon; Likhite,Shibi; Murtha,Matthew,J; Foust,Kevin,D; Rao,Meghan; Eagle,Amy; Kammesheidt,Anja; Christensen,Ashley; Mendell,Jerry,R; Burghes,Arthur,HM; Kaspar,Brian,K. 2011. Astrocytes from familial and sporadic ALS patients are toxic to motor neurons.  NATURE BIOTECHNOLOGY. Vol. 29, no. 9. (September): 824-U79.
  • Lioy,Daniel,T; Garg,Saurabh,K; Monaghan,Caitlin,E; Raber,Jacob; Foust,Kevin,D; Kaspar,Brian,K; Hirrlinger,Petra,G; Kirchhoff,Frank; Bissonnette,John,M; Ballas,Nurit; Mandel,Gail. 2011. A role for glia in the progression of Rett's syndrome.  NATURE. Vol. 475, no. 7357. (July): 497-U90.
  • Jukkola PI, Rogers JT, Kaspar BK, Weeber EJ, Nishijima I. 0. Secretin deficiency causes impairment in survival of neural progenitor cells in mice.  Hum Mol Genet.
  • Lin,Y; Roman,K; Foust,K,D; Kaspar,B,K; Bailey,M,T; Stephens,R,L. 2011. Glutamate Transporter GLT-1 Upregulation Attenuates Visceral Nociception and Hyperalgesia via Spinal Mechanisms Not Related to Anti-Inflammatory or Probiotic Effects.  Pain research and treatment. Vol. 2011, no. January: 507029-?.
  • Peterson JM, Kline W, Canan BD, Ricca DJ, Kaspar B, Delfín DA, Dirienzo K, Clemens PR, Robbins PD, Baldwin AS, Flood P, Kaumaya P, Frietas M, Kornegay JN, Mendell JR, Rafael-Fortney JA, Guttridge DC, Janssen PM. 2011. Peptide-based inhibition of NF-κB rescues diaphragm muscle contractile dysfunction in a murine model of Duchenne muscular dystrophy.  Molecular Medicine. Vol. 17, no. January: 508-515.
  • Bockstael,Olivier; Foust,Kevin,D; Kaspar,Brian; Tenenbaum,Liliane. 2011. Recombinant AAV delivery to the central nervous system.  Methods in molecular biology (Clifton, N.J.). Vol. 807, no. January: 159-177.
  • Bockstael, Foust, Kaspar, Tenenbaum. 2011. Recombinant AAV Delivery to the Central Nervous System. In Adeno Associated Virus Methods and Protocols. Edited by Snyder and Moullier. New York: Humana Press.
  • An MC; Lin W; Yang J; Dominguez B; Padgett D; Sugiura Y; Aryal P; Gould TW; Oppenheim RW; Hester ME; Kaspar BK; Ko CP; Lee KF. 2010. Acetylcholine negatively regulates development of the neuromuscular junction through distinct cellular mechanisms.  Proceedings Of The National Academy Of Sciences Of The United States Of America. Vol. 107, no. 23. (June): e10702.
  • Foust KD; Wang X; McGovern VL; Braun L; Bevan AK; Haidet AM; Le TT; Morales PR; Rich MM; Burghes AH; Kaspar BK. 2010. Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN.  Nature Biotechnology. Vol. 28, no. 3. (March): e271.
  • Sahenk Z, Galloway G, Edwards C, Malik V, Kaspar BK, Eagle A, Yetter B, Forgie A, Tsao D, Lin JC. 2010. TrkB and TrkC agonist antibodies improve function, electrophysiologic and pathologic features in Trembler J mice.  Exp Neurol. Vol. 224, no. 2. (January): 495-506.
  • Dodge JC, Treleaven CM, Fidler JA, Hester M, Haidet A, Handy C, Rao M, Eagle A, Matthews JC, Taksir TV, Cheng SH, Shihabuddin LS, Kaspar BK. 2010. AAV4-mediated expression of IGF-1 and VEGF within cellular components of the ventricular system improves survival in familial ALS mice.  Mol Ther. Vol. 18, no. 12. (January): 2075-2084.
  • Bevan AK, Hutchinson KR, Foust KD, Braun L, McGovern VL, Schmelzer L, Ward JG, Peturska JC, Lucchesi PA, Burghes AH, Kaspar BK. 2010. Early heart failure in the SMNDelta7 model of spinal muscular atrophy and correction by postnatal scAAV9-SMN delivery.  Hum Mol Genet. Vol. 19, no. 20. (January): 3895-3905.
  • Petruska JC, Kitay B, Boyce VS, Kaspar BK, Pearse DD, Gage FH, Mendell LM. 2010. Intramuscular AAV delivery of NT-3 alters synaptic transmission to motoneurons in adult rats.  Eur J Neurosci. Vol. 32, no. 6. (January): 997-1005.
  • Haidet AM, Mendell JR, Kaspar BK. 2010. Could gene therapy be the future for muscular dystrophy?.  Therapy. Vol. 7, no. 3. (January): 287-290.
  • Kaspar,Rita,Wen; Allen,Hugh,D; Ray,Will,C; Alvarez,Carlos,E; Kissel,John,T; Pestronk,Alan; Weiss,Robert,B; Flanigan,Kevin,M; Mendell,Jerry,R; Montanaro,Federica. 2009. Analysis of Dystrophin Deletion Mutations Predicts Age of Cardiomyopathy Onset in Becker Muscular Dystrophy.  CIRCULATION-CARDIOVASCULAR GENETICS. Vol. 2, no. 6. (December): 544-U51.
  • Kota Janaiah, Handy Chalonda R, Haidet Amanda M, Montgomery Chrystal L, Eagle Amy, Rodino-Klapac Louise R, Tucker Danielle, Shilling Christopher J, Therlfall Walter R, Walker Christopher M, Weisbrode Steven E, Janssen Paul ML, Clark Reed K, Sahenk Zarife, Mendell Jerry R, Kaspar Brian K. 2009. Follistatin Gene Delivery Enhances Muscle Growth and Strength in Nonhuman Primates.  Science. Vol. 1, no. 6. (November): e6ra15.
  • Hester M, Foust K, Kaspar RW, Kaspar BK. 2009. AAV as a gene transfer vector for the treatment of neurological disorders: Novel treatment thoughts for ALS.  Current Gene Therapy.
  • Hester,Mark,E; Foust,Kevin,D; Kaspar,Rita,W; Kaspar,Brian,K. 2009. AAV as a Gene Transfer Vector for the Treatment of Neurological Disorders: Novel Treatment Thoughts for ALS.  CURRENT GENE THERAPY. Vol. 9, no. 5. (October): 428-433.
  • Foust,; Chicoine,; Kaspar. 2009. The neonatal blood-brain barrier is functionally effective, and immaturity does not explain differential targeting of AAV9 Reply.  NATURE BIOTECHNOLOGY. Vol. 27, no. 9. (September): 805-805.
  • Kaspar RW, Wills CE, Kaspar BK. 2009. Gene therapy and informed consent decision making: nursing research directions.  Biol Res Nurs. Vol. 1, no. 11. (July): 98-107.
  • Kaspar,Rita,Wen; Allen,Hugh,D; Montanaro,Federica. 2009. Current understanding and management of dilated cardiomyopathy in Duchenne and Becker muscular dystrophy.  JOURNAL OF THE AMERICAN ACADEMY OF NURSE PRACTITIONERS. Vol. 21, no. 5. (May): 241-249.
  • Excoffon KJ, Koerber JT, Dickey DD, Murtha M, Keshavjee S, Kaspar BK, Zabner J, Schaffer DV. 2009. Directed evolution of adeno-associated virus to an infectious respiratory virus.  Proc Natl Acad Sci USA. Vol. 106, no. 10. (March): 3865-3870.
  • Rodino-Klapac L, Haidet AM, Kota J, Handy C, Kaspar BK, Mendell JR. 2009. Inhibition of myostatin with emphasis on follistatin as therapy for muscle disease.  Muscle Nerve. Vol. 39, no. January: 283-296.
  • Foust KD, Nurre E, Montgomery CL, Hernandez A, Chan CM, Kaspar BK. 2009. Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes.  Nat Biotechnol.. Vol. 27, no. Epub 2008 Dec 21. (January): 59-65.
  • Hester M, Song SW, Miranda C, Eagle A, Schwartz P, Kaspar BK. Two Factor Reprogramming of Human Neural Stem Cells into Pluripotency.  PLos One.
  • Brian K. Kaspar. 2009. RNA Interference Techniques. In Neuroscience Methods. Edited by Scott Q. Harper. Totowa, NJ: Humana/Springer Press.
  • Foust Kevin D, Kaspar Brian K. 2009. Over the barrier and through the blood to CNS delivery we go.  Cell Cycle. Vol. 8, no. 24. (January): 16-17.
  • Kaspar BK. 2008. Mesenchymal stem cells as trojan horses for GDNF delivery in ALS.  Mol Ther.. Vol. 16, no. 12. (December): 1905-1906.
  • Kim ML, Chandrasekharan K, Glass M, Shi S, Stahl MC, Kaspar B, Stanley P, Martin PT. 2008. O-fucosylation of muscle agrin determines its ability to cluster acetylcholine receptors.  Mol Cell Neurosci.. Vol. 39, no. 3. (November): 452-464.
  • Haidet, Amanda, M; Rizo, Liza; Handy, Chalonda; Umapathi, Priya; Eagle, Amy; Shilling, Chris; Boue, Daniel; Martin, Paul, T; Sahenk, Zarife; Mendell, Jerry, R; Kaspar, Brian K. 2008. Long-term enhancement of skeletal muscle mass and strength by single gene administration of myostatin inhibitors.  Proceedings of the National Academy of Sciences of the United States of America. Vol. 105, no. 11. (January): e4318.
  • Dodge, James, C; Haidet, Amanda, M; Yang, Wendy; Passini, Marco, A; Hester, Mark; Clarke, Jennifer; Roskelley, Eric, M; Treleaven, Christopher, M; Rizo, Liza; Martin, Heather; Kim, Soo, H; Kaspar, Rita; Taksir, Tatyana, V; Griffiths, Denise, A; Cheng, Seng, H; Shihabuddin, Lamya, S; Kaspar, Brian K. 2008. Delivery of AAV-IGF-1 to the CNS extends survival in ALS mice through modification of aberrant glial cell activity.  Molecular therapy : the journal of the American Society of Gene Therapy. Vol. 16, no. 6. (January): 1056-64.
  • Miller, Timothy, M; Smith, Richard, A; Kordasiewicz, Holly; Kaspar, Brian K. 2008. Gene-targeted therapies for the central nervous system.  Archives of neurology. Vol. 65, no. 4. (January): 447-51.
  • McCrate ME, Kaspar BK. 2008. Physical activity and neuroprotection in amyotrophic lateral sclerosis. Review.  Neuromolecular Med.. Vol. 10, no. 2. (January): 108-117.
  • Kaspar, Ray, Senut, Suhr, Gage. 2007. Ex Vivo and In Vivo Gene Delivery to the Brain. In Current Protocols in Human Genetics. n/a: n/a.
  • Rodino-Klapac LR, Chicoine LG, Kaspar BK, Mendell JR. 2007. Gene therapy for duchenne muscular dystrophy: expectations and challenges.  Archives of Neurology. Vol. 64, no. 9. (January): 1236-1241.
  • Miller, Timothy, M; Kim, Soo, H; Yamanaka, Koji; Hester, Mark; Umapathi, Priya; Arnson, Hannah; Rizo, Liza; Mendell, Jerry, R; Gage, Fred, H; Cleveland, Don, W; Kaspar, Brian K. 2006. Gene transfer demonstrates that muscle is not a primary target for non-cell-autonomous toxicity in familial amyotrophic lateral sclerosis.  Proceedings of the National Academy of Sciences of the United States of America. Vol. 103, no. 51. (December): 19546-19551.
  • Rizo,Liza; Shilling,Chris; Haidet,Amanda; Umapathi,Priya,Umapathi; Sahenk,Zarife; Mendell,Jerry,R; Kaspar,Brian,K. 2006. Recombinant AAV Gene Delivery of Follistatin for Muscle Enhancement in Models of Muscular Dystrophy.  MOLECULAR THERAPY. Vol. 13, no. May: S352-S352.
  • Dodge,James,C; Passini,Marco,A; Clarke,Jennifer; Grissett,Liza; Kim,Soo,Hyun; Wen,Rita; Taksir,Tatyana,V; Griffiths,Denise,A; Cheng,Seng,H; Kaspar,Brian,K; Shihabuddin,Lamya,S. 2006. Intracerebellar Injection of AAV-IGF-1 Improves Motor Function and Extends Survival in a Mouse Model of Amyotrophic Lateral Sclerosis.  MOLECULAR THERAPY. Vol. 13, no. May: S407-S407.
  • Haenggeli,Christine; Lepore,Angelo,C; Perez,Natalie; Kaspar,Brian,K; Rothstein,Jeffrey,D. 2006. Delivery of IGF-1 in the Spinal Cord of a Mouse Model of ALS after Intraparenchymal Injection of an Adeno-Associated Vector.  MOLECULAR THERAPY. Vol. 13, no. May: S94-S94.
  • Maheshri, Narendra; Koerber, James, T; Kaspar, Brian, K; Schaffer, David V. 2006. Directed evolution of adeno-associated virus yields enhanced gene delivery vectors.  Nature biotechnology. Vol. 24, no. 2. (February): 198-204.
  • Kaspar BK and Gage FH. 2006. Gene Therapy and Neurological Disorders. In Gene Therapy. Edited by Pedro Lowenstein. San Diego: Alden International, Taylor and Francis Books.
  • Kaspar BK. 2006. Neural repair: regeneration and functional recovery: Gene therapy: direct viral delivery. In New Encyclopedia of Neuroscience. Edited by Larry Squire. n/a: n/a.
  • Koerber, James, T; Maheshri, Narendra; Kaspar, Brian, K; Schaffer, David V. 2006. Construction of diverse adeno-associated viral libraries for directed evolution of enhanced gene delivery vehicles.  Nature protocols. Vol. 1, no. 2. (January): 701-706.
  • Borrell, Victor; Kaspar, Brian, K; Gage, Fred, H; Callaway, Edward M. 2006. In vivo evidence for radial migration of neurons by long-distance somal translocation in the developing ferret visual cortex.  Cerebral cortex (New York, N.Y. : 1991). Vol. 16, no. 11. (January): 1571-1583.
  • Lee GK, Maheshri N, Kaspar BK, Schaffer DV. 2005. PEG Conjugation Mederately Protects Adeno-Associated Viral Vectors Against Antibody Neutralization.  Biotechnology and Bioengineering. Vol. 92, no. 1. (October): 24-34.
  • Kaspar, Brian, K; Frost, Lindsay, M; Christian, Lindsey; Umapathi, Priya; Gage, Fred H. 2005. Synergy of insulin-like growth factor-1 and exercise in amyotrophic lateral sclerosis.  Annals of neurology. Vol. 57, no. 5. (May): 649-655.
  • Miller, Timothy, M; Kaspar, Brian, K; Kops, Geert, J; Yamanaka, Koji; Christian, Lindsey, J; Gage, Fred, H; Cleveland, Don W. 2005. Virus-delivered small RNA silencing sustains strength in amyotrophic lateral sclerosis.  Annals of neurology. Vol. 57, no. 5. (January): 773-776.
  • Kaspar, Brian, K; Roth, David, M; Lai, N Chin; Drumm, Jeffrey, D; Erickson, Dawn, A; McKirnan, M Dan; Hammond, H Kirk. 2005. Myocardial gene transfer and long-term expression following intracoronary delivery of adeno-associated virus.  The journal of gene medicine. Vol. 7, no. 3. (January): 316-324.
  • Hsieh, Jenny; Aimone, James, B; Kaspar, Brian, K; Kuwabara, Tomoko; Nakashima, Kinichi; Gage, Fred H. 2004. IGF-I instructs multipotent adult neural progenitor cells to become oligodendrocytes.  The Journal of cell biology. Vol. 164, no. 1. (January): 111-122.
  • Kaspar, Brian, K; Llado, Jeronia; Sherkat, Nushin; Rothstein, Jeffrey, D; Gage, Fred H. 2003. Retrograde viral delivery of IGF-1 prolongs survival in a mouse ALS model. Cited 186 times.  Science (New York, N.Y.). Vol. 301, no. 5634. (August): 839-842.
  • South, Samantha, M; Kohno, Tatsuro; Kaspar, Brian, K; Hegarty, Deborah; Vissel, Bryce; Drake, Carrie, T; Ohata, Megumi; Jenab, Shirzad; Sailer, Andreas, W; Malkmus, Shelle; Masuyama, Takashi; Horner, Philip; Bogulavsky, Johanna; Gage, Fred, H; Yaksh, Tony, L; Woolf, Clifford, J; Heinemann, Stephen, F; Inturrisi, Charles E. 2003. A conditional deletion of the NR1 subunit of the NMDA receptor in adult spinal cord dorsal horn reduces NMDA currents and injury-induced pain.  The Journal of neuroscience : the official journal of the Society for Neuroscience. Vol. 23, no. 12. (June): 5031-5040.
  • Lai, Karen; Kaspar, Brian, K; Gage, Fred, H; Schaffer, David V. 2003. Sonic hedgehog regulates adult neural progenitor proliferation in vitro and in vivo. Cited 168 times.  Nature neuroscience. Vol. 6, no. 1. (January): 21-27.
  • Lie, D Chichung; Dziewczapolski, Gustavo; Willhoite, Andrew, R; Kaspar, Brian, K; Shults, Clifford, W; Gage, Fred H. 2002. The adult substantia nigra contains progenitor cells with neurogenic potential. Cited 145 times.  The Journal of neuroscience : the official journal of the Society for Neuroscience. Vol. 22, no. 15. (August): 6639-6649.
  • Kaspar, Brian, K; Vissel, Bryce; Bengoechea, Tasha; Crone, Steven; Randolph-Moore, Lynne; Muller, Rolf; Brandon, Eugene, P; Schaffer, David; Verma, Inder, M; Lee, Kuo-Fen; Heinemann, Stephen, F; Gage, Fred H. 2002. Adeno-associated virus effectively mediates conditional gene modification in the brain. Cited 41 times.  Proceedings of the National Academy of Sciences of the United States of America. Vol. 99, no. 4. (February): 2320-2325.
  • Kaspar, Brian, K; Erickson, Dawn; Schaffer, David; Hinh, Linda; Gage, Fred, H; Peterson, Daniel A. 2002. Targeted retrograde gene delivery for neuronal protection. Cited 55 times.  Molecular therapy : the journal of the American Society of Gene Therapy. Vol. 5, no. 1. (January): 50-56.
  • Kaspar,R,L. 2002. Integrating Internet assignments into a biochemistry/molecular biology laboratory course.  BIOCHEMISTRY AND MOLECULAR BIOLOGY EDUCATION. Vol. 30, no. 1. (January): 36-39.
  • Palmer, T, D; Schwartz, P, H; Taupin, P; Kaspar, B; Stein, S, A; Gage, F H. 2001. Cell culture. Progenitor cells from human brain after death. Cited 170 times.  Nature. Vol. 411, no. 6833. (May): 42-43.
  • Senut, M, C; Suhr, S, T; Kaspar, B; Gage, F H. 2000. Intraneuronal aggregate formation and cell death after viral expression of expanded polyglutamine tracts in the adult rat brain.  The Journal of neuroscience : the official journal of the Society for Neuroscience. Vol. 20, no. 1. (January): 219-229.
  • Ueba, T; Kaspar, B; Zhao, ; XGage, F H. 1999. Repression of human fibroblast growth factor 2 by a novel transcription factor.  The Journal of biological chemistry. Vol. 274, no. 15. (April): 10382-10387.
  • Donahue BA, McArthur JG, Spratt SK, Bohl D, Lagarde C, Sanchez l, Kaspar BK, Sloan BA, Lee YL, Danos O, Snyder RO. 1999. Selective uptake and sustained expression of AAV vectors following subcutaneous delivery.  J Gene Med. Vol. 1, no. 1. (February): 31-42.
  • Snyder, R, O; Spratt, S, K; Lagarde, C; Bohl, D; Kaspar, B; Sloan, B; Cohen, L, K; Danos, O. 1997. Efficient and stable adeno-associated virus-mediated transduction in the skeletal muscle of adult immunocompetent mice.  Human gene therapy. Vol. 8, no. 16. (November): 1891-1900.
  • Song SW, Miranda CJ, Braun L, Meyer K, Frakes AE, Ferraiuolo L, Likhite L, Bevan AK, Foust KD McConnell MJ, Walker CM, Kaspar BK. Sustained expression of MHC class I protects motor neurons from ALS astrocyte-induced toxicity.  Nature Medicine.

Awards, Honors and Organizations

  • Fellow of the American Association for the Advancement of Science (AAAS), 2013
Nationwide Children's Hospital
700 Children's Drive Columbus, Ohio 43205 614.722.2000