Gene Therapy That Works

2013/08/22 by Inder M. Verma · 1 citation
Biochemistry, Genetics and Molecular Biology · Medicine · #Virus-based gene therapy research #CRISPR and Genetic Engineering #CAR-T cell therapy research

paper · doi:10.1126/science.1242551

openalex publication_date 2013/08/22 · openalex created_date 2016/06/24 · openalex updated_date 2026/08/02

Abstract

Gene therapy trials show a beneficial effect in children suffering from a neurodegenerative disorder or an immunodeficiency disease. [Also see Research Articles by Biffi et al. and Aiuti et al. ]

Cited by

• Genetic disorder [wikipedia]

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