Demitri de la Cruz

Optimization of base editors for the functional correction of SMN2 as a treatment for spinal muscular atrophy

Christiano R. R. Alves, Leillani L. Ha, Rebecca Yaworski et al. · Nature Biomedical Engineering · 2023 · 42 citations · Full text

Medicine, Neurology, Neuroscience +3

An Engineered Adeno-Associated Virus Capsid Mediates Efficient Transduction of Pericytes and Smooth Muscle Cells of the Brain Vasculature

Servio H. Ramirez, Jonathan F. Hale, Siobhán McCarthy et al. · Human Gene Therapy · 2023 · 24 citations · Full text

Pharmacy, Bioengineering, Biomedical Engineering +14

Base editing as a genetic treatment for spinal muscular atrophy

Christiano R. R. Alves, Leillani L. Ha, Rebecca Yaworski et al. · bioRxiv (Cold Spring Harbor Laboratory) · 2023 · 19 citations · Full text

Life Sciences, Genetics, Molecular Genetics +17

An AAV capsid increases transduction of striatum and a ChAT promoter allows selective cholinergic neuron transduction

Miguel C. Santoscoy, Paula Espinoza, Demitri de la Cruz et al. · Molecular Therapy — Methods & Clinical Development · 2023 · 13 citations · Full text

Life Sciences, Neurobiology, Neuroscience +17

In vivo selection in non-human primates identifies AAV capsids for on-target CSF delivery to spinal cord

Killian S. Hanlon, Ming Cheng, Roberto Montoro Ferrer et al. · Molecular Therapy · 2024 · 12 citations · Full text

Pharmacy, Molecular Medicine, Drug Delivery +14