Last Posted: Sep 27, 2019
- Advances and highlights in primary immunodeficiencies in 2017.
Chinen Javier et al. The Journal of allergy and clinical immunology 2018 142(4) 1041-1051 - Gene therapy for hemophilia: Progress to date and challenges moving forward.
Gollomp Kandace L et al. Transfusion and apheresis science : official journal of the World Apheresis Association : official journal of the European Society for Haemapheresis 2019 Aug - Advancing Gene-Targeted Therapies for Central Nervous System Disorders: Proceedings of a Workshop
NASEM, Workshop Proceedings, September 20, 2019 - Future Preventive Gene Therapy of Polygenic Diseases from a Population Genetics Perspective
RT Oliynyk, BioRXIV, September 18, 2019 - Current Status on Clinical Development of Adeno-Associated Virus-Mediated Liver-Directed Gene Therapy for Inborn Errors of Metabolism.
Ginocchio Virginia Maria et al. Human gene therapy 2019 Sep - Successful engraftment of gene-corrected hematopoietic stem cells in non-conditioned patients with Fanconi anemia.
Río Paula et al. Nature medicine 2019 Sep 25(9) 1396-1401 - Update on gastric cancer treatments and gene therapies.
Biagioni Alessio et al. Cancer metastasis reviews 2019 Sep - CRISPR-Edited Stem Cells in a Patient with HIV and Acute Lymphocytic Leukemia.
Xu Lei et al. The New England journal of medicine 2019 Sep - First gene therapy for β-thalassemia approved
C Harrison, Nature Biotechnology, September 9, 2019 - Gene Therapy for Choroideremia-Progress and Remaining Questions.
Duncan Jacque L et al. JAMA ophthalmology 2019 Aug
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