CRISPR-Based Therapeutic Genome Editing: Strategies and In Vivo Delivery by AAV Vectors.
review · Level V
Where this comes from
- Record sourced from PubMed, PMID 32243786.
- Also identified by DOI 10.1016/j.cell.2020.03.023 and PMC identifier 7236621.
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Abstract
The development of clustered regularly interspaced short-palindromic repeat (CRISPR)-based biotechnologies has revolutionized the life sciences and introduced new therapeutic modalities with the potential to treat a wide range of diseases. Here, we describe CRISPR-based strategies to improve human health, with an emphasis on the delivery of CRISPR therapeutics directly into the human body using adeno-associated virus (AAV) vectors. We also discuss challenges facing broad deployment of CRISPR-based therapeutics and highlight areas where continued discovery and technological development can further advance these revolutionary new treatments.
Medical subject headings
- CRISPR-Cas Systems
- Dependovirus
- Gene Editing
- Genetic Therapy
- Genetic Vectors