In this issue:
CRISPR-Cas9 in vivo gene editing for transthyretin amyloidosis
Triplet CF therapy superior to single/doublet for heterozygous Phe508del genotypes
Real-world effectiveness of elexacaftor/tezacaftor/ivacaftor for CF
Omidubicel better than umbilical cord blood transplantation for blood cancers
Neoadjuvant T-VEC reduces risk of resectable melanoma recurrence
Biodistribution of onasemnogene abeparvovec in human tissue
Bepirovirsen demonstrates preliminary antiviral efficacy for chronic HBV
CARAMBA: first clinical trial of advanced Sleeping Beauty transposon technology
Infrastructure changes for implementation of gene therapy for haemophilia in Europe
Patient preferences for gene therapy in haemophilia
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