In this issue:
Sense suppression of BCL11A shows potential as treatment for sickle cell disease
CRISPR-Cas9 gene editing for sickle cell disease and β-thalassemia
Voretigene neparvovec-rzyl in RPE65 mutation–associated inherited retinal dystrophy
Cost effectiveness of voretigene neparvovec in Germany
Prognostic model for the prediction of neurotoxicity following CAR T-cell therapy
MOXIe demonstrates neurological function improvements with omaveloxolone for Friedreich ataxia
Nusinersen treatment in adults with SMA type 3
Nusinersen may improve function and motor stabilisation in type 2 & 3 SMA
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