Therapeutic In Vivo Gene Editing Achieved by a Hypercompact CRISPR‐Cas12f1 System Delivered with All‐in‐One Adeno‐Associated Virus (Adv. Sci. 19/2024)
Source abstract
Abstract
CRISPR Gene Therapy In article number 2308095, Wei Li, Fei Teng, and colleagues introduce a hypercompact CRISPR-Cas12f1 system characterized by enhanced gene editing capabilities and minimal off-target effects. Applied to the treatment of inherited retinal diseases, this system shows significant therapeutic outcomes, highlighting its substantial promise for gene therapy applications.
Source: openalex_inverted_index · Quality: baseline
Research context
OpenAlex topics: CRISPR and Genetic Engineering · Virus-based gene therapy research · CAR-T cell therapy research
SciVX discovery tags are terminology-based. OpenAlex topics are source-provided classifications.
References
Reference metadata was not supplied for this record.