The realization of CRISPR gene therapy

The realization of CRISPR gene therapy

The inaugural CRISPR-based drug Casgevy has been approved by several medical agencies, with other CRISPR-based therapies currently in clinical trials. Although there are technological hurdles to overcome, chemical biology has a vital role in developing recent breakthroughs in base editing, prime edi...

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Veröffentlicht in:Nature chemical biology 2024-07, Vol.20 (7), p.791-795
Hauptverfasser: Ma, Yitong, Qi, Lei S.
Format: Artikel
Sprache:eng
Schlagworte:
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Biochemical Engineering
Biochemistry
Biology
Bioorganic Chemistry
Bone marrow
Cancer therapies
Cell Biology
Chemistry
Chemistry and Materials Science
Chemistry/Food Science
Clinical trials
Comment
CRISPR
Disease
Editing
Epigenetics
FDA approval
Gene therapy
Genetic disorders
Genetic engineering
Hemoglobin
Hepatitis B
HIV
Human immunodeficiency virus
Lymphocytes
Muscular dystrophy
Mutation
Sickle cell anemia
Viral infections
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Beschreibung
Zusammenfassung:The inaugural CRISPR-based drug Casgevy has been approved by several medical agencies, with other CRISPR-based therapies currently in clinical trials. Although there are technological hurdles to overcome, chemical biology has a vital role in developing recent breakthroughs in base editing, prime editing and epigenetic editing into future treatments.
ISSN:1552-4450
1552-4469
1552-4469
DOI:10.1038/s41589-024-01645-x