A5042530758- Muscle Physiology and Disorders
- Virus-based gene therapy research
- Endoplasmic Reticulum Stress and Disease
- Poxvirus research and outbreaks
- Neurogenesis and neuroplasticity mechanisms
- Monoclonal and Polyclonal Antibodies Research
- Viral Infectious Diseases and Gene Expression in Insects
- CRISPR and Genetic Engineering
- Bacterial Genetics and Biotechnology
- Immunodeficiency and Autoimmune Disorders
- Tissue Engineering and Regenerative Medicine
- Neuroinflammation and Neurodegeneration Mechanisms
- Enzyme Structure and Function
- Multiple Sclerosis Research Studies
The Ohio State University
2021-2023
Nationwide Children's Hospital
2021
National Cancer Institute
2012
In a phase 1/2, open-label dose escalation trial, we delivered rAAVrh74.MCK.GALGT2 (also B4GALNT2) bilaterally to the legs of two boys with Duchenne muscular dystrophy using intravascular limb infusion. Subject 1 (age 8.9 years at dosing) received 2.5 × 1013 vector genome (vg)/kg per leg (5 vg/kg total) and subject 2 6.9 5 (1 1014 total). No serious adverse events were observed. Muscle biopsy evaluated 3 or 4 months post treatment versus baseline showed evidence GALGT2 gene expression...
RpoS, the master sigma factor during stationary phase and under a variety of stress conditions, is regulated at multiple levels, including degradation. Degradation dependent upon ClpXP RssB adaptor protein. H-NS, nucleoid-associated protein, affects degradation RpoS; in absence RpoS stable. The mechanisms involved this regulation were not known. We have found that H-NS inhibits expression iraD iraM, genes coding for two antiadaptor proteins stabilize when overexpressed. by iraM independent...
We have examined the effects of intravenous (IV) delivery rAAVrh74.MHCK7. GALGT2 in golden retriever muscular dystrophy (GRMD) model Duchenne Muscular Dystrophy (DMD). After baseline testing, GRMD dogs were treated at 3 months age and reassessed 6 months. This 3–6 month range is a period rapid disease progression, thus offering relatively short window to establish treatment efficacy. Measures analyzed included muscle AAV transduction, transgene expression, -induced glycosylation, pathology,...