Immune responses to AAV vectors: overcoming barriers to successful gene therapy
Mingozzi F, High KA. Immune responses to AAV vectors: overcoming barriers to successful gene therapy. Blood 2013;122:23-36 Abstract Gene…
Impact of AAV Capsid-Specific T-Cell Responses on Design and Outcome of Clinical Gene Transfer Trials with Recombinant Adeno-Associated Viral Vectors: An Evolving Controversy
Ertl HCJ, High KA. Impact of AAV Capsid-Specific T-Cell Responses on Design and Outcome of Clinical Gene Transfer Trials with…
Use of home telemedicine for critical illness rehabilitation: an Indian success story
Unnikrishnan D, Raman D, Joshi D, Ajaikumar BS. Use of home telemedicine for critical illness rehabilitation: an Indian success story.…
CRISPR-cas gene-editing as plausible treatment of neuromuscular and nucleotide-repeat-expansion diseases: A systematic review
Babacic H, Mehta A, Merkel O, Schoser B. CRISPR-cas gene-editing as plausible treatment of neuromuscular and nucleotide-repeat-expansion diseases: A systematic…
Targeting angiogenesis in Duchenne muscular dystrophy
Podkalicka P, Mucha O, Dulak J, Loboda A. Targeting angiogenesis in Duchenne muscular dystrophy. Cellular and molecular life sciences :…
Frequency of reported pain in adult males with muscular dystrophy
Jacques MF, Stockley RC, Bostock EI, Smith J, DeGoede CG, Morse CI. Frequency of reported pain in adult males with…
Skeletal disproportion in glucocorticoid-treated boys with Duchenne muscular dystrophy
Kao KT, Joseph S, Capaldi N, et al. Skeletal disproportion in glucocorticoid-treated boys with Duchenne muscular dystrophy. European journal of…
Analysis of Different Device Interactions in a Virtual Reality Task in Individuals With Duchenne Muscular Dystrophy-A Randomized Controlled Trial
de Freitas BL, da Silva TD, Crocetta TB, et al. Analysis of Different Device Interactions in a Virtual Reality Task…
Population Pharmacokinetics of Vamorolone (VBP15) in Healthy Men and Boys With Duchenne Muscular Dystrophy
Mavroudis PD, van den Anker J, Conklin LS, et al. Population Pharmacokinetics of Vamorolone (VBP15) in Healthy Men and Boys…
Patient views on research use of clinical data without consent: Legal, but also acceptable?
Richter G, Borzikowsky C, Lieb W, Schreiber S, Krawczak M, Buyx A. Patient views on research use of clinical data…
