AAV9.LAMP2B gene therapy shows clinical benefit in Danon disease
1. In seven patients with Danon disease, a single infusion of adeno-associated virus serotype 9 carrying transgene LAMP2B (AVV9.LAMP2B) therapy ...
Tag: gene therapy
#VisualAbstract: Gene Therapy with Fidanacogene Elaparvovec in Adults with Hemophilia B is Superior to Prophylaxis
Click here to read this study in NEJM. ©2024 2 Minute Medicine, Inc. All rights reserved. No works may be reproduced ...
Taysha Gene Therapies: TSHA-102 Gene Therapy Improves Quality of Life in Patients with Rett Syndrome
A patient who received TSHA-102 had durable improvements in motor skills, communication, and socialization. TSHA-102 was well tolerated with no ...
Intrathecal gene therapy as a treatment for giant axonal neuropathy
1. In this dose-escalation study, using intrathecal gene transfer with scAAV9/JeT-GAN demonstrated potential improvement in motor function. 2. Adverse events ...
Pfizer: Beqvez Gene Therapy Decreases Bleeding Rates in Hemophilia B Patients
Hemophilia B patients given Beqvez had a decreased incidence of total bleeding Beqvez reduced the use of routine factor IX ...
Gene therapy cost-effective for treating sickle cell disease in simulation models
1. In this cost analysis, compared to standard care, it was found that gene therapy for sickle cell disease (SCD) ...
KLKB1 gene editing therapy reduces kallikrein levels in hereditary angioedema
1. In this randomized controlled trial, gene-editing therapy targeting the kallikrein B1 gene (KLKB1) led to a significant reduction in ...
#VisualAbstract: Gene therapy improves bilirubin levels in Crigler-Najjar syndrome
Click here to read the study in NEJM ©2023 2 Minute Medicine, Inc. All rights reserved. No works may be reproduced ...
Gene therapy improves bilirubin levels in Crigler-Najjar syndrome
1. In this randomized controlled trial, a high dose of GNT0003 gene therapy was associated with decreased serum bilirubin and ...
Etranacogene dezaparvovec gene therapy reduces bleeding in severe hemophilia B
1. Etranacogene dezaparvovec gene therapy was more effective than prophylactic factor IX infusion at reducing annualized bleeding rate amongst patients ...