Cargando…

THE PROMISE AND CHALLENGE OF THERAPEUTIC GENOME EDITING

Genome editing, involving precise manipulation of cellular DNA sequences to alter cell fates and organism traits, offers the potential to both understand human genetics and cure genetic disease as never before. Scientific, technical and ethical aspects of employing CRISPR technology for therapeutic...

Descripción completa

Detalles Bibliográficos
Autor principal: Doudna, Jennifer A.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8992613/
https://www.ncbi.nlm.nih.gov/pubmed/32051598
http://dx.doi.org/10.1038/s41586-020-1978-5
Descripción
Sumario:Genome editing, involving precise manipulation of cellular DNA sequences to alter cell fates and organism traits, offers the potential to both understand human genetics and cure genetic disease as never before. Scientific, technical and ethical aspects of employing CRISPR technology for therapeutic applications in humans are discussed, focusing on specific examples that highlight both opportunities and challenges. Genome editing is or will soon be in the clinic for several diseases, with more applications in the pipeline. The rapid pace of the field demands active efforts to ensure responsible use of this breakthrough technology to treat, cure and prevent genetic disease.