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Nature Biomedical Engineering (2)
Gene Therapy (1)

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Showing 1–3 of 3 results

Advanced filters: Author: Demitri de la Cruz Clear advanced filters

Treatment of a severe vascular disease using a bespoke CRISPR–Cas9 base editor in mice

Engineering a mutant-specific customized base editor precisely corrects a mutation while minimizing bystander edits, leading to substantial phenotypic recovery in mouse models of multisystemic smooth muscle dysfunction syndrome.

Christiano R. R. Alves
Sabyasachi Das
Patricia L. Musolino
Research11 Sept 2025
Nature Biomedical Engineering

P: 1-16
Optimization of base editors for the functional correction of SMN2 as a treatment for spinal muscular atrophy

Optimized base editors targeting the exon-7 mutation in SMN2 restore expression of the survival motor neuron (SMN) protein to normal levels, as shown in mice with spinal muscular atrophy and in fibroblasts from patients with this genetic disease.

Christiano R. R. Alves
Leillani L. Ha
Benjamin P. Kleinstiver
Research06 Dec 2023
Nature Biomedical Engineering

Volume: 8, P: 118-131
Engineered AAV capsids mediate transduction of murine neurofibroma and sciatic nerve

Edwina Abou Haidar
Shilpa Prabhakar
Casey A. Maguire
Research10 Jun 2025
Gene Therapy

Volume: 32, P: 385-397