Gene Therapy Research Review, Issue 2

In this issue:

Autologous ex vivo lentiviral gene therapy shows promise for ADA-SCID
Oncolytic HSV immunovirotherapy may be a viable salvage option for paediatric brain tumours
Lumasiran, an RNA interference therapeutic for primary hyperoxaluria type 1
Risdiplam in type 1 spinal muscular atrophy
KarMMA demonstrates efficacy of CAR T cell immunotherapy in relapsed/refractory myeloma
Lumacaftor–ivacaftor safe and effective for paediatric patients with F508del-CFTR cystic fibrosis
CRISPR base editing may be suitable for a large proportion of patients with recessive IRD
Retinal gene therapy for CNGA3-mutated achromatopsia

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