Abstract

We propose to evaluate the safety of the gene therapy in Limb girdle muscular dystrophy, specifically alpha-sarcoglycan deficiency, using a recombinant, replication-deficient, adeno-associated viral delivery system. The primary objective of this study is to determine the safety of adeno-associated virus (AAV) delivered via needle injection into the extensor digitorum (EDB) muscle. The EDB is a small foot muscle without important function. Muscles on both feet will be injected. One foot will receive the alpha-sarcoglycan gene and the other an injection of sterile saline. This will be done in a double-blind manner without the investigators or the patient's knowledge of which side is receiving the gene vs placebo. Six weeks after injection both EDB muscles will biopsied to determine if the gene has been effective in replacing the missing protein. This will be the first human trial of gene therapy for any type of muscular dystrophy. Currently there is no medication that is known to be effective for the treatment of any of the forms of LGMD. If this study is successful, it will represent the first step in a series of studies directed more toward efficacy of gene therapy in this disease.

  Funding Agency

Agency
National Institute of Health (NIH)
Institute
National Center for Research Resources (NCRR)
Type
General Clinical Research Centers Program (M01)
Project #
5M01RR000034-40
Application #
6404451
Study Section
National Center for Research Resources Initial Review Group (RIRG)
Project Start
1976-12-01
Project End
2001-11-30
Budget Start
Budget End
Support Year
40
Fiscal Year
2000
Total Cost
Indirect Cost

  Institution

Name
Ohio State University
Department
Type
DUNS #
098987217
City
Columbus
State
OH
Country
United States
Zip Code
43210

  Publications

Beversdorf, David Q; Carpenter, Allen L; Alexander, Jessica K et al. (2018) Influence of Serotonin Transporter SLC6A4 Genotype on the Effect of Psychosocial Stress on Cognitive Performance: An Exploratory Pilot Study. Cogn Behav Neurol 31:79-85
Tita, Alan T N; Lai, Yinglei; Landon, Mark B et al. (2017) Predictive Characteristics of Elevated 1-Hour Glucose Challenge Test Results for Gestational Diabetes. Am J Perinatol 34:1464-1469
Criado, Kristen K; Sharp, William G; McCracken, Courtney E et al. (2017) Overweight and obese status in children with autism spectrum disorder and disruptive behavior. Autism :1362361316683888
Lee, Kyoung Suk; Lennie, Terry A; Yoon, Ju Young et al. (2017) Living Arrangements Modify the Relationship Between Depressive Symptoms and Self-care in Patients With Heart Failure. J Cardiovasc Nurs 32:171-179
Landon, Mark B; Grobman, William A; Eunice Kennedy Shriver National Institute of Child Health and Human Development Maternal–Fetal Medicine Units Network (2016) What We Have Learned About Trial of Labor After Cesarean Delivery from the Maternal-Fetal Medicine Units Cesarean Registry. Semin Perinatol 40:281-6
Blackwell, Sean C; Landon, Mark B; Mele, Lisa et al. (2016) Relationship Between Excessive Gestational Weight Gain and Neonatal Adiposity in Women With Mild Gestational Diabetes Mellitus. Obstet Gynecol 128:1325-1332
Navari, Rudolph M; Qin, Rui; Ruddy, Kathryn J et al. (2016) Olanzapine for the Prevention of Chemotherapy-Induced Nausea and Vomiting. N Engl J Med 375:134-42
Salazar, Ashley; Tolivaisa, Susan; Allard, Donna et al. (2016) What we have learned about best practices for recruitment and retention in multicenter pregnancy studies. Semin Perinatol 40:321-7
Harper, Lorie M; Mele, Lisa; Landon, Mark B et al. (2016) Carpenter-Coustan Compared With National Diabetes Data Group Criteria for Diagnosing Gestational Diabetes. Obstet Gynecol 127:893-8
Palatnik, Anna; Mele, Lisa; Landon, Mark B et al. (2015) Timing of treatment initiation for mild gestational diabetes mellitus and perinatal outcomes. Am J Obstet Gynecol 213:560.e1-8

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