In Vivo Genome Engineering for the Treatment of Muscular Dystrophies
Sign Up to like & getrecommendations! Published in 2020 at "Current Stem Cell Reports"
DOI: 10.1007/s40778-020-00173-3
Abstract: Purpose of Review Muscular dystrophies (MDs) are a heterogeneous collection of inherited disorders which cause progressive muscle loss and weakness/hypotonia. Owing to the genetic root of MDs, CRISPR/Cas9 genome editing has been investigated as a… read more here.
Keywords: crispr; crispr cas9; vivo genome; muscular dystrophies ... See more keywords
In Vivo Genome Editing Partially Restores Alpha1-Antitrypsin in a Murine Model of AAT Deficiency.
Sign Up to like & getrecommendations! Published in 2018 at "Human gene therapy"
DOI: 10.1089/hum.2017.225
Abstract: CRISPR (clustered regularly interspaced short palindromic repeats) genome editing holds promise in the treatment of genetic diseases that currently lack effective long-term therapies. Patients with alpha-1 antitrypsin (AAT) deficiency develop progressive lung disease due to… read more here.
Keywords: genome editing; crispr; vivo genome; model ... See more keywords
In vivo genome-wide CRISPR screening in murine acute myeloid leukemia uncovers microenvironmental dependencies
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DOI: 10.1182/bloodadvances.2022007250
Abstract: Key Points In vivo CRISPR screens in AML define key interactors of the microenvironment, including integrins, immune modulators, and glycosylation. Eight in vivo–specific hits are recurrently associated with adverse prognosis: BTBD6, FERMT3, ILK, SLC19A1, TAP2,… read more here.
Keywords: crispr; screening murine; genome wide; wide crispr ... See more keywords