| SELECTED PUBLICATIONS |
| Rodino-Klapac LR, Montgomery CL, Bremer WG, Shontz KM, Malik V, Davis N, Sprinkle S, Campbell KJ, Sahenk Z, Clark KR, Walker CM, Mendell JR, Chicoine LG. Persistent expression of FLAG-tagged micro dystrophin in nonhuman primates following intramuscular and vascular delivery. Mol Ther. 2010 Jan;18(1):109-17. PubMed ID: 19904237 |
| Saechao C, Valles-Ayoub Y, Esfandiarifard S, Haghighatgoo A, No D, Shook S, Mendell JR, Rosales-Quintero X, Felice KJ, Morel CF, Pietruska M, Darvish D.Novel GNE Mutations in Hereditary Inclusion Body Myopathy Patients of Non-Middle Eastern Descent. Genet Test Mol Biomarkers. 2010 Jan 10 . |
Flanigan, K. M., D. M. Dunn, A. von Niederhausern, P. Soltanzadeh, E. Gappmaier, M. T. Howard, J. B. Sampson, J. R. Mendell, C. Wall, W. M. King, A. Pestronk, J. M. Florence, A. M. Connolly, K. D. Mathews, C. M. Stephan, K. S. Laubenthal, B. L. Wong, P. J. Morehart, A. Meyer, 2009. Mutational spectrum of DMD mutations in dystrophinopathy patients: application of modern diagnostic techniques to a large cohort. Hum Mutat 30:1657-66. PubMed ID: 19937601 |
Flanigan, K. M., D. M. Dunn, A. von Niederhausern, M. T. Howard, J. Mendell, A. Connolly, C. Saunders, A. Modrcin, M. Dasouki, G. P. Comi, R. Del Bo, A. Pickart, R. Jacobson, R. Finkel, L. Medne, and R. B. Weiss. 2009. DMD Trp3X nonsense mutation associated with a founder effect in North American families with mild Becker muscular dystrophy. Neuromuscul Disord 19:743-8. PubMed ID: 19793655 |
Johnson, P. R., B. C. Schnepp, J. Zhang, M. J. Connell, S. M. Greene, E. Yuste, R. C. Desrosiers, and K. R. Clark. 2009. Vector-mediated gene transfer engenders long-lived neutralizing activity and protection against SIV infection in monkeys. Nat Med 15:901-6. PubMed ID: 19448633 |
Kota, J., R. R. Chivukula, K. A. O'Donnell, E. A. Wentzel, C. L. Montgomery, H. W. Hwang, T. C. Chang, P. Vivekanandan, M. Torbenson, K. R. Clark, J. R. Mendell, and J. T. Mendell. 2009. Therapeutic microRNA delivery suppresses tumorigenesis in a murine liver cancer model. Cell 137:1005-17. PubMed ID: 19524505 |
Martin, P. T., R. Xu, L. R. Rodino-Klapac, E. Oglesbay, M. Camboni, C. L. Montgomery, K. Shontz, L. G. Chicoine, K. R. Clark, Z. Sahenk, J. R. Mendell, and P. M. Janssen. 2009. Overexpression of Galgt2 in skeletal muscle prevents injury resulting from eccentric contractions in both mdx and wild-type mice. Am J Physiol Cell Physiol 296:C476-88. PubMed ID: 19109526 |
Mendell, J. R., L. R. Rodino-Klapac, X. Rosales-Quintero, J. Kota, B. D. Coley, G. Galloway, J. M. Craenen, S. Lewis, V. Malik, C. Shilling, B. J. Byrne, T. Conlon, K. J. Campbell, W. G. Bremer, L. Viollet, C. M. Walker, Z. Sahenk, and K. R. Clark. 2009. Limb-girdle muscular dystrophy type 2D gene therapy restores alpha-sarcoglycan and associated proteins. Ann Neurol 66:290-7. PubMed ID: 19798725 |
Rodino-Klapac, L. R., C. L. Montgomery, W. G. Bremer, K. M. Shontz, V. Malik, N. Davis, S. Sprinkle, K. J. Campbell, Z. Sahenk, K. R. Clark, C. M. Walker, J. R. Mendell, and L. G. Chicoine. 2009. Persistent Expression of FLAG-tagged Micro dystrophin in Nonhuman Primates Following Intramuscular and Vascular Delivery. Mol Ther. PubMed ID: 19904237 |
Schnepp, B. C., R. L. Jensen, K. R. Clark, and P. R. Johnson. 2009. Infectious molecular clones of adeno-associated virus isolated directly from human tissues. J Virol 83:1456-64.
PubMed ID: 19019948 |
Butterfield, R. J., D. Ramachandran, S. J. Hasstedt, B. E. Otterud, M. F. Leppert, K. J. Swoboda, and K. M. Flanigan. 2009. A novel form of juvenile recessive ALS maps to loci on 6p25 and 21q22. Neuromuscul Disord 19:279-87. PubMed ID: 19318250 |
Gurvich, O. L., B. Maiti, R. B. Weiss, G. Aggarwal, M. T. Howard, and K. M. Flanigan. 2009. DMD exon 1 truncating point mutations: amelioration of phenotype by alternative translation initiation in exon 6. Hum Mutat 30:633-40. PubMed ID: 19206170 |
Maiti, B., S. Arbogast, V. Allamand, M. W. Moyle, C. B. Anderson, P. Richard, P. Guicheney, A. Ferreiro, K. M. Flanigan, and M. T. Howard. 2009. A mutation in the SEPN1 selenocysteine redefinition element (SRE) reduces selenocysteine incorporation and leads to SEPN1-related myopathy. Hum Mutat 30:411-6. PubMed ID: 19067361 |
| Clark, K. R., and S. T. Walsh Dec 2009, posting date. Crystal structure of a 3B3 variant--a broadly neutralizing HIV-1 scFv antibody 2009/09/29. PubMed ID: 19785005 |
| Mendell JR, Rodino-Klapac LR, Rosales-Quintero X, Kota J, Coley BD, Galloway G, Craenen JM, Lewis S, Malik V, Shilling C, Byrne BJ, Conlon T, Campbell KJ, Bremer WG, Viollet L, Walker CM, Sahenk Z, Clark KR.Limb-girdle muscular dystrophy type 2D gene therapy restores alpha-sarcoglycan and associated proteins. Ann Neurol. 2009 Sep;66(3):290-7. PubMed ID: 19798725 |
| Chicoine LG, Stewart JA Jr, Lucchessi PA. Is Reseveratrol the magic bullet for pulmonary hypertension? PubMed ID: 19597034 |
| Elsheikh B, Kissel JT, Christoforidis G, Wicklund M, Kehagias DT, Chiocca EA, Mendell JR. Spinal angiography and epidural venography in juvenile muscular atrophy of the distal arm "Hirayama disease". Muscle Nerve. 2009 Aug;40(2):206-12.PMID: 19609908 PubMed ID: 19609908 |
| Elsheikh B, Prior T, Zhang X, Miller R, Kolb SJ, Moore D, Bradley W, Barohn R, Bryan W, Gelinas D, Iannaccone S, Leshner R, Mendell JR, Mendoza M, Russman B, Smith S, King W, Kissel JT. An analysis of disease severity based on SMN2 copy number in adults with spinal muscular atrophy. Muscle Nerve. 2009 Oct;40(4):652-6. PubMed ID: 19760790 |
| Flanigan KM, Dunn DM, von Niederhausern A, Soltanzadeh P, Gappmaier E, Howard MT, Sampson JB, Mendell JR, Wall C, King WM, Pestronk A, Florence JM, Connolly AM, Mathews KD, Stephan CM, Laubenthal KS, Wong BL, Morehart PJ, Meyer A, Finkel RS, Bonnemann CG, Medne L, Day JW, Dalton JC, Margolis MK, Hinton VJ; United Dystrophinopathy Project Consortium, Weiss RB.Mutational spectrum of DMD mutations in dystrophinopathy patients: application of modern diagnostic techniques to a large cohort. Hum Mutat. 2009 Dec;30(12):1657-66. PubMed ID: 19937601 |
| Foust KD, Nurre E, Montgomery CL, Hernandez A, Chan CM, Kaspar BK. Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes. Nat Biotechnol. 2009 Jan;27(1):59-65 PubMed ID: 19098898 |
| Guenther UP, Handoko L, Varon R, Stephani U, Tsao CY, Mendell JR, Lützkendorf S, Hübner C, von Au K, Jablonka S, Dittmar G, Heinemann U, Schuetz A, Schuelke M.Clinical variability in distal spinal muscular atrophy type 1 (DSMA1): determination of steady-state IGHMBP2 protein levels in five patients with infantile and juvenile disease. J Mol Med. 2009 Jan;87(1):31-41. PubMed ID: 18802676 |
| Kaspar RW, Allen HD, Ray WC, Alvarez CE, Kissel JT, Pestronk A, Weiss RB, Flanigan KM, Mendell JR, Montanaro F. Analysis of dystrophin deletion mutations predicts age of cardiomyopathy onset in becker muscular dystrophy. Circ Cardiovasc Genet. 2009 Dec;2(6):544-51. PubMed ID: 20031633 |
| Kota J, Chivukula RR, O'Donnell KA, Wentzel EA, Montgomery CL, Hwang HW, Chang TC, Vivekanandan P, Torbenson M, Clark KR, Mendell JR, Mendell JT. Therapeutic microRNA delivery suppresses tumorigenesis in a murine liver cancer model. Cell. 2009 Jun 12;137(6):1005-17. PubMed ID: 19524505 |
| Martin PT, Xu R, Rodino-Klapac LR, Oglesbay E, Camboni M, Montgomery CL, Shontz K, Chicoine LG, Clark KR, Sahenk Z, Mendell JR, Janssen PM.Overexpression of Galgt2 in skeletal muscle prevents injury resulting from eccentric contractions in both mdx and wild-type mice. Am J Physiol Cell Physiol. 2009 Mar;296(3):C476-88. PubMed ID: 19109526 |
| Rodino-Klapac LR, Haidet AM, Kota J, Handy C, Kaspar BK, Mendell JR. Inhibition of myostatin with emphasis on follistatin as a therapy for muscle disease. Muscle Nerve. 2009 Mar;39(3):283-96. Review. PubMed ID: 19208403 |
| S.Q. Harper. Progress and challenges in RNAi therapy for Huntington's Disease. (2009) Archives of Neurology |
| Thrush PT, Allen HD, Viollet L, Mendell JR. Re-examination of the electrocardiogram in boys with Duchenne muscular dystrophy and correlation with its dilated cardiomyopathy. Am J Cardiol. 2009 Jan 15;103(2):262-5. PubMed ID: 19121448 |
| Tsao CY, Mendell JR. Partial epilepsy in an adolescent male with limb-girdle muscular dystrophy 1B. J Child Neurol. 2009 Mar;24(3):346-8. PubMed ID: 19258295 |
| Viollet L, Gailey S, Thornton DJ, Friedman NR, Flanigan KM, Mahan JD, Mendell JR. Utility of cystatin C to monitor renal function in Duchenne muscular dystrophy. Muscle Nerve. 2009 Sep;40(3):438-42. PubMed ID: 19623638 |
| Miller TM, Smith RA, Kordasiewicz H, Kaspar BK. Gene-targeted therapies for the central nervous system. Arch Neurol. 2008 Apr;65(4):447-51. Epub 2008 Feb 11 PubMed ID: 18268183 |
| McCrate ME, Kaspar BK. Physical activity and neuroprotection in amyotrophic lateral sclerosis. Neuromolecular Med. 2008;10(2):108-17. Epub 2008 Feb 20 PubMed ID: 18286388 |
| Haidet AM, Rizo L, Handy C, Umapathi P, Eagle A, Shilling C, Boue D, Martin PT, Sahenk Z, Mendell JR, Kaspar BK. Long-term enhancement of skeletal muscle mass and strength by single gene administration of myostatin inhibitors. Proc Natl Acad Sci U S A. 2008 Mar 18;105(11):4318-22. Epub 2008 Mar 11 PubMed ID: 18334646 |
Gurvich, O.L., Tuohy, T.M., Howard, M.T., Finkel, R.S., Medne, L., Anderson, C.B., Weiss, R.B., Wilton, S.D., Flanigan, K.M. (2008) DMD pseudoexon mutations: splicing efficiency, phenotype, and potential therapy. Ann Neurol., 2008 Jan;63(1):81-9. PubMed ID: 18059005 |
J.L. McBride*, R.L. Boudreau*, S.Q. Harper* (shared first authorship), A. Mas Monteys, P.D. Staber, I. Martins, B. Gilmore, H. Burstein, R.W. Peluso, B. Polisky, B.J. Carter, and B.L. Davidson. MicroRNA shuttles mitigate short-hairpin RNA mediated toxicity in the brain: Implications for therapeutic development of RNA interference. (2008) Proceedings of the National Academy of Sciences, USA 105(15): 5868-73. PubMed ID: 18398004 |
| Sahenk Z, Oblinger J, Edwards C. Neurotrophin-3 deficient Schwann cells impair nerve regeneration. Exp Neurol. 2008 Aug;212(2):552-556. Epub 2008 Apr 23. PubMed ID: 18511043 |
| A. Packer, Y. Xing, S.Q. Harper, L. Jones, and B.L. Davidson. The bi-functional microRNA mir9/mir9* regulates REST and coREST and is down-regulated in Huntington's desease (2008) Journal of Neuroscience, 28(53):14341-6 PubMed ID: 19118166 |
| Brian K Kaspar, Mesenchymal Stem Cells as Trojan Horses for GDNF Delivery in ALS. Molecular Therapy, Vol. 16(12):2008. PubMed ID: 19032270 |
| Calvert, Thomas; Chicoine, Louis; Liu, Yusen; Nelin, Leif. Deficiency of Mitogen Activated Protein Kinase Phosphatase-1 Results in iNOS-mediated Hypotension in Response to Low-Dose Endotoxin. Am J Physiol Heart Circ Physiol. 2008 Apr; 294(4): H1621-9 PMID: 182813281 PubMed ID: 182813281 |
| Haidet AM, Rizo L, Handy C, Umapathi P, Mendell JR, Kaspar BK, et. al. Long-term enhancement of skeletal muscle mass and strength by single gene administration of myostatin inhibitors. Proc Natl Acad Sci U S A. 2008 Mar 18;105(11):4318-22. PubMed ID: 18334646 |
| Ishihara A., Shields K.M., Litsky A.S., Mattoon J.S., Weisbrode S.E., Bartlett J.S., and Bertone A.L. Osteogenic gene regulation and relative acceleration of healing by adenoviral-mediated transfer of human BMP-2 or -6 in equine osteotomy and ostectomy models. J Orthop Res. 2008 Jun;26(6):764-71. [PMID: 18241059] PubMed ID: 18241059 |
| Kannanayakal TJ, Mendell JR, Kuret J. Casein kinase 1 alpha associates with the tau-bearing lesions of inclusion body myositis. Neurosci Lett. 2008 Jan 31;431(2):141-5. PubMed ID: 18191026 |
| Kim ML, Chandrasekharan K, Glass M, Shi S, Stahl MC, Kaspar B, Stanley P, Martin PT. O-funocylation of muscle agrin determines its ability to cluster acetylcholine receptors, Mol Cell Neurosci, 2008 Nov;39(3):452-64 PubMed ID: 18775496 |
| Martin PT, Xu R, Rodino-Klapac LR, Oglesbay E, Camboni M, Montgomery CL, Shontz K, Chicoine L, Clark KR, Sahenk Z, Mendell JR, Janssen PM. Overexpression of Galgt2 in skeletal muscle prevents injury resulting from eccentric contractions in both mdx and wild type mice. Am J Physiol Cell Physiol. 2008 PMID: 19109526 [PubMed – as supplied by publisher] 2008 Dec 24. [Epub ahead of print] PubMed ID: 19109526 |
| Rodino-Klapac LR, Lee J-S, Mulligan RC, Clark KR, Mendell JR. Lack of toxicity of alpha-sarcoglycan overexpression supports clinical gene transfer in LGMD2D. Neurology 2008 Jul 22;71(4):240-7. PubMed ID: 18525034 |
| Rosales XQ, Shilling C, Wall C, Chu M-L, Mendell JR. Fidelity of Serum Gamma-glutamyl transferase in differentiating skeletalmuscle from liver disease in Duchenne muscular dystrophy. J Child Neurol. 2008 Jul;23(7):748-51. PubMed ID: 18354148 |
| Rossana Chang, Louis G. Chicoine, Hongmei Cui, Nancy L. Kanagy, Benjimen R. Walker, Yusen Liu, B. Keith English, and Leif D. Nelin. Cytokine-induced Arginase Activity in Pulmonary Endothelial Cells is Dependent on Src-Family Tyrosine Kinase Activity. Am J Physiol Lung Cell Mol Physiol . 2008 Oct;295(4):L688-L697. PMID: 18621907 PubMed ID: 18621907 |
| S.Q. Harper and Gonzalez-Alegre P. Lentivirus-mediated RNA interference in mammalian neurons. (2008) Methods in Molecular Biology 442, 95-112. PubMed ID: 18369781 |
| Sen S., Conroy S., Hynes S.O., McMahon J., O'Doherty A., Bartlett J.S., Akhtar Y., Adegbola T., Connolly C.E., Sultan S., Barry F., Katusic Z.S., O'Brien T. Gene delivery to the vasculature mediated by low-titre adeno-associated virus serotypes 1 and 5. J Gene Med. 2008 Feb;10(2):143-51.[PMID: 18067196] PubMed ID: 18067196 |
| Stachler, M.D., Chen, I., Ting, A.Y., and Bartlett, J.S. Site-specific modification of AAV vector particles with biophysical probes and targeting ligands using biotin ligase. Mol Ther. 2008 Jun 17 [Epub ahead of print]. [PMID: 18560418] PubMed ID: 18560418 |
| Stenger M, Rose M, Joshi M, Rogers L, Chicoine LG, Welty S, Bauer J, Nelin LD, Inhaled Nitric Oxide Prevents 3-nitrotyrosine Formation in the Lungs of Neonatal Mice Exposed to Lethal Hyperoxia. Am J Physiol Lung Cell Mol Physiol. 2008.(submitted) |
| Wagner KR, Fleckenstein JL, Amato AA, Barohn RJ, Mendell JR et al. A phase I/IItrial of MYO-029 in adult subjects with muscular dystrophy. Ann Neurol. 2008 May;63(5):561-71. PubMed ID: 18335515 |
| Zaiss, A.K., Cotter, M.J., White, L.R., Clark, S.A., Wong, C.W., Holers, V.M., Bartlett, J.S., and Muruve, D.A. Complement is an essential component of the immune response to adeno-associated virus vectors. J Virol. 2008 Mar;82(6):2727-40. Epub 2008 Jan 16. [PMID: 18199646] PubMed ID: 18199646 |
| Zhang X, Chow, CY, Sahenk Z, Shy ME, Meisler MH, Li, J. Mutation of FIG4 causes a rapidly progressive, asymmetric neuronal degeneration. Brain. 2008 Jun 12. PubMed ID: 18556664 |
Rodino-Klapac LR, Chicoine LG, Kaspar BK, Mendell JR. Gene therapy for duchenne muscular dystrophy: expectations and challenges.
Arch Neurol. 2007 Sep; 64( 9): 1236-41. PubMed ID: 17846262 |
Rodino-Klapac L, Chicoine L, Kaspar B, Mendell JR. Gene Therapy for Duchenne Muscular Dystrophy: Expectations and Challenges. Arch Neurol. 2007 Sep;64(9):1236-41. PubMed ID: 17846262 |
King WM, Ruttencutter R, Nagaraja HN, Matkovic V, Landoll J, Hoyle C, Mendell JR, Kissel JT. Orthopedic outcomes of long-term daily corticosteroid treatment in Duchenne muscular dystrophy. Neurology. 2007 May 8;68(19):1607-13. PubMed ID: 17485648 |
Rodino-Klapac L, Janssen PML, Montgomery CL, Coley BD, Chicoine LG, Clark KR, Mendell JR. A Translational Approach for Limb Vascular Delivery of the Micro-dystrophin Gene without High Volume or High Pressure for Treatment of Duchenne Muscular Dystrophy. J Transl Med. 2007 Sep 24;5:45. PubMed ID: 17892583 |
Xu, R, Camboni, M., Martin, P.T., Postnatal overexpression of the CT GalNAc transferase inhibits muscular dystrophy in mdx mice without altering muscle growth or neuromuscular development: Evidence for a utrophin-independent mechanism. Neuromuscul Disord. 2007 Feb. 12; [Epub ahead of print] PubMed ID: 17300937 |
| Fu, H., Kang, L., Jennings, J.S., Moy, S.S., Perez, A., DiRosario, J., McCarty, D.M., Muenzer, J., Significantly increased lifespan and improved behavioral performances by rAAV gene delivery in adult Mucopolysaccharidosis IIIB mice. Gene Therapy, 2007 Apr 26; [Epub ahead of print] PubMed ID: 17460717 |
| Arthur J Pope, Lawrence Druhan, Jorge E Guzman, Scott P Forbes, Velayutham Murugesan, Deqin Lu, Yong Xia, Louis G Chicoine, Narasimham L. Parinandi, and Arturo Juan Cardounel. Role of DDAH-1 in Lipid Peroxidation Product mediated inhibition of Endothelial NO Generation. Am J Physiol Cell Physiol 293: C1679 - C1686. E-pub Sep 19, 2007; doi:10.1152/ajpcell.00224.2007 |
| Louise R Rodino-Klapac, Paul ML Janssen, Chrystal L Montgomery, Brian D Coley, Louis G Chicoine, K Reed Clark, and Jerry R Mendell. A translational approach for limb vascular delivery of the micro-dystrophin gene without high volume or high pressure for treatment of Duchenne muscular dystrophy. Journal of Translational Medicine 2007, 5:45 doi:10.1186/1479-5876-5-45 |
| Nelin LD, Stenger MR, Malleske DT, Chicoine LG. Vascular Arginase and Hypertension. Current Hypertension Reviews. 3:2007 (in press) |
| Zachos, T., Diggs, A., Weisbrode, S., Bartlett, J.S., and Bertone, A. Mesenchymal stem cell-mediated gene delivery of bone-morphogenic protein-2 in an articular fracture model. Mol Ther. Epub 2007 May [doi:10.1038/sj.mt.6300192] 2007. |
| Martin, P.T. Congenital muscular dystrophy – Glycosylation takes center stage. Nature Clinical Practice Neurology. 2006 Apr; 2 (4) 222-230. PubMed ID: 16932553 |
| Shi, W., Hemminiki, A., and Bartlett, J.S. Capsid modifications overcome low heterogeneous expression of heparan sulfate proteoglycan that limits AAV2-mediated gene transfer and therapeutic efficacy in human ovarian carcinoma. Gyn. Oncol. 67(7):1145-55. 2006. PubMed ID: 16870238 |
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Choi VW, McCarty DM, Samulski RJ. Host cell DNA repair pathways in adeno-associated viral genome processing. J Virol. 2006 Nov;80(21):10346-56.
PubMed ID: 16445862 |
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Storek B, Harder NM, Banck MS, Wang C, McCarty DM, Janssen WG, Morrison JH, Walsh CE, Beutler AS. Intrathecal long-term gene expression by self-complementary adeno-associated virus type 1 suitable for chronic pain studies in rats. Mol Pain. 2006 Jan 30;2(1):4 [Epub ahead of print]
PubMed ID: 17041215 |
P. Allaire, B. Ritter, S. Thomas, J. Burman, A. Denisov, V. Legendre-Guillmen, S.Q. Harper, B.L. Davidson, K. Gehring, and P. McPherson. Connecdenn, A DENN domain-containing protein functioning in synaptic vescle endocytosis. (2006) Journal of Neuroscience 26(51) 13202-13212 PubMed ID: 17182770 |
| Sahenk Z. Neurotrophins and peripheral neuropathies. Brain Pathol. 2006 Oct;16(4):311-9. PubMed ID: 17107601 |
| Arnold, G.S., Sasser, A.K., Stachler, M., and Bartlett, J.S. Metabolic biotinylation provides a unique platform for the purification and targeting of multiple AAV vector serotypes. Mol Ther. 14(1): 97-106. 2006. PubMed ID: 16624620 |
| Borras, T., Xue, W., Choi, V.W., Samulski, R.J., Bartlett, J.S., Li, G., and Chisolm, S.S. Mechanisms of rAAV transduction in Glaucoma-Associated Human Trabecular Meshwork Cells. J Gene Med. 8:589-602. 2006. PubMed ID: 16506246 |
| Ishihara, A., Bertone, A., Zachos, T.A, and Bartlett, J.S. Relative permissiveness and cytotoxicity of equine chondrocytes, synovial cells, and bone marrow derived mesenchymal stem cells to adenoviral vector gene delivery. Amer J Vet Res. 67(7):1145-55. 2006. PubMed ID: 16817735 |
| Maheshri N, Koerber JT, Kaspar BK, Schaffer DV. Directed evolution of adeno-associated virus yields enhanced gene delivery vectors.Nature Biotechnology, 2006. Feb 24; 198-204. PubMed ID: 16429148 |
| Mendell JR, Clark KR (2006). Challenges for gene therapy for muscular dystrophy. Curr neurol Neurosci Rep. 6(1):47-56. PubMed ID: 16469271 |
| Mendell, J.R., Boue, D., and Martin, P.T. The congenital muscular dystrophies: recent advances and insights. Pediatr Dev Pathol, 2006 Nov-Dec; 9 (6): 427-43 PubMed ID: 17163796 |
| S.Q. Harper, P.D. Staber, C.R. Beck, S.K. Fineberg, C.S. Stein, D. Ochoa, and B.L. Davidson. Optimization of feline immunodeficiency viral vectors for RNA interference. (2006), Journal of Virology 80(19), 9371-9380 PubMed ID: 16973543 |
| Sharif, F., Hynes, S.O., McMahon, J., Cooney, R., Conroy, S., Dockery, P., Duffy, G., Crowley, J., Kieran, D., Bartlett, J.S., and O’Brien, T. Gene-eluting stents: A comparison of adenoviral and adeno-associated viral gene delivery to the blood vessel wall in vivo. Human Gene Ther. 17(7): 741-750. 2006 PubMed ID: 16839273 |
| Shi, X., Fang, G., Shi, W., and Bartlett, J.S. Insertional mutagenesis at positions 520 and 584 of the adeno-associated virus type 2 (AAV2) capsid gene and generation of AAV2 vectors with eliminated heparin-binding ability and introduced novel tropism. Human Gene Ther 17(3):353-361. 2006. PubMed ID: 16544984 |
| Stachler, M., and Bartlett, J.S. Mosaic vectors comprised of modified AAV1 capsid proteins for efficient vector purification and targeting to vascular endothelial cells. Gene Ther. 13:926-931. 2006. PubMed ID: 16482202 |
| Timothy M. Miller, Soo H. Kim, Mark Hester, Priya Umapathi, Hannah Arnson, Lisa Rizo, Jerry Mendell, Fred H. Gage, Don W. Cleveland, Brian K. Kaspar. Gene transfer demonstrates that muscle is not a primary target for non-cell-autonomous toxicity in familial amyotrophic lateral sclerosis. Proc Natl Acad Sci U S A. 2006 Dec 19;103(51):19546-51. Epub 2006 Dec 12. PubMed ID: 17164329 |
| Nelin LD, LG Chicoine, KM Reber, BK English, TL Young and Y Liu. Cytokine-induced endothelial arginase expression is dependent on epidermal growth factor receptor. Am J Respir Cell Mol Biol, 33(4):394-401, 2005. Epub 2005 Jun 30 PubMed ID: 15994432 |
Choi VW, Samulski RJ, McCarty DM. Effects of adeno-associated virus DNA hairpin structure on recombination. J Virol. 2005 June; 79 (11): 6801-7 PubMed ID: 15890919 |
Hacker UT, Wingenfeld L, Kofler DM, Schuhmann NK, Lutz S, Herold T, King SB, Gerner FM, Perabo L, Rabinowitz J, McCarty DM, Samulski RJ, Hallek M, Buning H. Adeno-associated virus serotypes 1 to 5 mediated tumor cell directed gene transfer and improvement of transduction efficiency. J Gene Med. 2005 Nov; 7 (11): 1429-38 PubMed ID: 15945124 |
Sahenk Z, Nagaraja H, McCracken BS, King WM, Freimer ML, Cedarbaum JM, Mendell JR: NT-3 promotes nerve regeneration and sensory improvement in CMT1A mouse models and in patients. Neurology 2005; 65(5):681-689. PubMed ID: 16157899 |
| Martin, P.T., The dystroglycanopathies: the new disorders of O-linked glycosylation. Semin. Pediatr. Neurol., 2005 Sep; 12:152-8. PubMed ID: 16584074 |
| B.L. Davidson and S.Q. Harper. Viral delivery of short hairpin RNAs. (2005) Methods in Enzymology 392, 145-173. PubMed ID: 15644180 |
| Chen CL, Jensen RL, Schnepp BC, Connell MJ, Shell R, Sferra TJ, Bartlett JS, Clark KR, Johnson PR (2005). Molecular characterization of adeno-associated viruses infection children. Journal of Virology 79(23):14781-92. PubMed ID: 16282478 |
| Chen, C-L., Jensen, R., Schnepp, B.C., Connell, M.J., Shell, R, Sferra, T.J., Bartlett, J.S., Clark, K.R., and Johnson, P.R. Molecular Characterization of Adeno-Associated Viruses Infecting Children. J Virol. 79: 14781-14792. 2005. PubMed ID: 16282478 |
| Dent, K.M., Dunn, D.M., von Niederhausern, A.C., Aoyagi, A.T., Kerr, L., Bromberg, M.B., Tuohy, T., White, S., den Dunnen, J.T., Weiss, R.B., and Flanigan, K.M. (2005) Improved molecular diagnosis of dystrophinopathies in an unselected clinical cohort. Am J Med Genetics 134A:295-298. PubMed ID: 15723292 |
| Howard, M.T., Aggarwal, G., Anderson, C.B., Khatri, S., Flanigan, K.M., Atkins, J.F. (2005) Recoding elements located adjacent to a subset of eukaryal selenocysteine-specifying UGA codons. EMBO J., Mar 24. PubMed ID: 15791204 |
| Johnson PR, Schnepp BC, Connell MJ, Rohne D, Robinson S, Krivulka GR, Lord CI, Zinn R, Montefiori DC, Letvin NL, and Clark KR (2005). Novel Adeno-Associated Virus Vector Vaccine Restricts Replication of Simian Immunodeficiency Virus in Macaques. Journal of Virology 79, 955–965. PubMed ID: 15613324 |
| Johson PR, Schnepp BC, Connell MJ, Rohne D, Robinson S, Krivulka GR, Lord CI, Zinn R, Montefiori DC, Letvin NL, Clark KR (2005). Novel adeno-associated virus vector vaccine restricts replication of simian immunodeficiency virus in macaques. Journal of Virology 79(2):955-65. PubMed ID: 15613324 |
| Kaspar, BK, Frost LM, Christian L, Umapathi P, Gage FH. Synergy of Insulin-like Growth Factor-1 and Exercise in Amyotrophic Lateral Sclerosis. Annals of Neurology. 2005 May 57 649-655. PubMed ID: 15852403 |
| Lawson, V.H., Graham, B.V., and Flanigan, K.M. (2005) Clinical and electrophysiologic features of CMT2A with novel mutations in the Mfn2 Gene. Neurology, 65:197-204. PubMed ID: 16043786 |
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