Pablo Morales

RETRACTED ARTICLE: Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN

Kevin D. Foust, Xueyong Wang, Vicki L. McGovern et al.|Nature Biotechnology|2010

Cited by 690Open Access

Systemic Gene Delivery in Large Species for Targeting Spinal Cord, Brain, and Peripheral Tissues for Pediatric Disorders

Adam K. Bevan, Sandra Duqué, Kevin D. Foust et al.|Molecular Therapy|2011

Cited by 331Open Access

Improving Single Injection CSF Delivery of AAV9-mediated Gene Therapy for SMA: A Dose–response Study in Mice and Nonhuman Primates

Kathrin Meyer, Laura Ferraiuolo, Leah Schmelzer et al.|Molecular Therapy|2014

Cited by 276Open Access

A BMP/activin A chimera is superior to native BMPs and induces bone repair in nonhuman primates when delivered in a composite matrix

Howard Seeherman, Stephen P. Berasi, Christopher Brown et al.|Science Translational Medicine|2019

Cited by 66

of the BMP-2/absorbable collagen sponge (ACS) concentration approved for clinical use. Initial toxicology studies were negative. These results support evaluations of BV-265/CM as an alternative to BMP-2/ACS in clinical trials for orthopedic conditions requiring augmented healing.

Gene Therapy Corrects Brain and Behavioral Pathologies in CLN6-Batten Disease

Jacob T. Cain, Shibi Likhite, Katherine A. White et al.|Molecular Therapy|2019

Cited by 58Open Access

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