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. 2023 Mar 17;15(6):1813. doi: 10.3390/cancers15061813

Table 2.

CRISPR in clinical practice.

Status Name/Trial Trade Name Disease Reference
2017 FDA approved Voretigene neparvovec Luxterna Retinal dystrophy [43,44]
2019 FDA approved Onasemnogene abeparvovec Zolgensma SMA (Pediatric Patients, < 2 y/o) [45,46]
Currently evaluated in clinical trials Lovotibeglogene autotemcel LentiGlobin BB305 SCD, Thalassemia, TDT [47]
Currently evaluated in clinical trials CLIMB THAL-111, CLIMB SCD-111 - SCD, TDT [48]