Last Posted: Jun 11, 2021
- Gene therapy helps children with immunodeficiency
O'Leary, Nature Medicine, June 10, 2021
- Gene Therapy for Mucopolysaccharidosis Type II-A Review of the Current Possibilities.
Zapolnik Pawel et al. International journal of molecular sciences 2021 22(11)
- RPE65-associated inherited retinal diseases: consensus recommendations for eligibility to gene therapy.
Sodi Andrea et al. Orphanet journal of rare diseases 2021 16(1) 257
- A Review of Acquired Autoimmune Blistering Diseases in Inherited Epidermolysis Bullosa: Implications for the Future of Gene Therapy.
Patel Payal M et al. Antibodies (Basel, Switzerland) 2021 10(2)
- Cost-effectiveness of a hypothetical cell or gene therapy cure for sickle cell disease.
Salcedo Jonathan et al. Scientific reports 2021 11(1) 10838
- Updated review of therapeutic strategies for Charcot-Marie-Tooth disease and related neuropathies.
Pisciotta Chiara et al. Expert review of neurotherapeutics 2021
van Spronsen Francjan J et al. Nature reviews. Disease primers 2021 7(1) 36
- Safety and improved efficacy signals following gene therapy in childhood blindness caused by GUCY2D mutations.
Jacobson Samuel G et al. iScience 2021 24(5) 102409
- ‘N of 1’ therapies need a better model
AA Rus, Nature Medicine, May 24, 2021
- Partial recovery of visual function in a blind patient after optogenetic therapy
JA Sahel et al, Nature Medicine, May 24, 2021
- Gene Therapy: A Possible Alternative to CFTR Modulators?
Mercier J et al. Frontiers in pharmacology 2021 12648203
- Selecting β-thalassemia Patients for Gene Therapy: A Decision-making Algorithm.
Baronciani Donatella et al. HemaSphere 2021 5(5) e555
- Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency.
Kohn Donald B et al. The New England journal of medicine 2021 5
- Financial considerations in expanded access policy for gene therapies: A tough nut to crack?
Polak Tobias B et al. Molecular therapy : the journal of the American Society of Gene Therapy 2021
- Optimizing language for effective communication of gene therapy concepts with hemophilia patients: a qualitative study.
Hart Daniel P et al. Orphanet journal of rare diseases 2021 16(1) 189
- Current Status of Gene Therapy Research in Polyglutamine Spinocerebellar Ataxias.
Afonso-Reis Ricardo et al. International journal of molecular sciences 2021 22(8)
- Gene therapy may not be as expensive as people think: challenges in assessing the value of single and short-term therapies.
Garrison Louis P et al. Journal of managed care & specialty pharmacy 2021 27(5) 674-681
- Hematopoietic stem cell transplantation for people with β-thalassaemia.
Sharma Akshay et al. The Cochrane database of systematic reviews 2021 4CD008708
- Recent Advancements in the Diagnosis and Treatment of Leukodystrophies.
Bradbury Allison M et al. Seminars in pediatric neurology 2021 37100876
- Spinal Muscular Atrophy.
Nicolau Stefan et al. Seminars in pediatric neurology 2021 37100878
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