(From the February 2016 Issue of MedStat)
A number of possible immunotherapies for cancer are based on T-cells that can target and kill tumors, but tumors that downregulate HLA class I evade cytotoxic T lymphocytes (CTL). Researchers at Nationwide Children’s Hospital recently characterized the expression of HLA class I and its required associated β-2-microglobulin (B2M) moleculein 18 different types of pediatric solid tumors, proposing a “CTL target score” to predict tumor susceptibility to T-cell based therapies. Kellie Haworth, MD, a research fellow in the Division of Hematology, Oncology and Blood and Marrow Transplant at Nationwide Children’s, is the lead author of the study, which was published in the journal Pediatric Blood & Cancer.
Read the Pediatrics Online article.
Nationwide Children’s Hospital Researchers Receive Cystic Fibrosis Grant to Study Cell Therapy
A team of physician-scientists at Nationwide Children’s Hospital, led by Susan D. Reynolds, PhD, principal investigator in the Center for Perinatal Research in The Research Institute, recently received a grant of more than $150,000 from the Cystic Fibrosis Foundation to study the safety and effectiveness of cell therapy for the treatment of cystic fibrosis. The grant will help researchers identify the optimal cell for use as cell therapy, focusing specifically on the airway tissue stem cell, which self-renews and is the source of all cells covering the surface of the lung’s airways
Learn more in the News Room release.
Mouse Model of Duchenne Muscular Dystrophy Reflects Small Population, but Could Provide Big Payoff
Duplication of exons, the parts of a gene that encode amino acids, cause six to 10 percent of cases of Duchenne muscular dystrophy (DMD), which is due to mutations in the DMD gene. Researchers at Nationwide Children’s Hospital have developed the first mouse model to investigate whether skipping, or excluding, one copy of a duplicated exon during assembly of the final gene message may have a large payoff: production of normal dystrophin, the missing protein that protects muscle from injury, and eventual elimination of DMD symptoms. Kevin Flanigan, MD, principal investigator in the Center for Gene Therapy in The Research Institute and neurologist at Nationwide Children’s, is lead author of the study, which was published in the journal Neuromuscular Disorders.
Read the Pediatrics Nationwide article.