Crystal, Ronald G.

Safety of Direct Intraparenchymal AAVrh.10-Mediated Central Nervous System Gene Therapy for Metachromatic Leukodystrophy

Rosenberg, J. B., Chen, A., De, B. P., Dyke, J. P., Ballon, D. J., Monette, S., Ricart Arbona, R. J., Kaminsky, S. M., Crystal, R. G., & Sondhi, D. (2021) · Human Gene Therapy

Virus-based gene therapy researchLysosomal Storage Disorders Research

Cited by 41

Genetic Modification of the Lung Directed Toward Treatment of Human Disease

Sondhi, D., Stiles, K. M., De, B. P., & Crystal, R. G. (2017) · Human Gene Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 36

AAVrh.10 delivery of novel APOE2-Christchurch variant suppresses amyloid and Tau pathology in alzheimer's disease mice

Günaydin, C., Sondhi, D., Kaminsky, S. M., Lephart, H. C., Leopold, P. L., Hackett, N. R., Khanna, R., & Crystal, R. G. (2024) · Molecular Therapy

Alzheimer's disease research and treatmentsVirus-based gene therapy research

Cited by 26

Prevalence of the Apolipoprotein E Arg145Cys Dyslipidemia At-Risk Polymorphism in African-Derived Populations

Abou Ziki, M. D., Strulovici-Barel, Y., Hackett, N. R., Rodriguez-Flores, J. L., Mezey, J. G., Salit, J., Radisch, S., Hollmann, C., Chouchane, L., Malek, J., et al. (2014) · The American Journal of Cardiology

Lipoproteins and Cardiovascular HealthLipid metabolism and disorders

Cited by 21

365. Long-Term Toxicology Evaluation of AAVrh. 10hARSA Administration to the CNS of Nonhuman Primates to Treat Metachromatic Leukodystrophy

Sondhi, D., Rosenberg, J. B., De, B. P., Monette, S., Chen, A., Flagiello, T., Caffrey, E., Kaminsky, S. M., Aubourg, P., & Crystal, R. G. (2016) · Molecular Therapy

Lysosomal Storage Disorders ResearchVirus-based gene therapy research

Cited by 3