Last Posted: Dec 21, 2020
- Annual Academy of Sickle Cell and Thalassaemia (ASCAT) conference: a summary of the proceedings.
Strunk Crawford et al. BMC proceedings 2020 Dec 14(Suppl 20) 21 - Immune Reconstitution After Gene Therapy Approaches in Patients With X-Linked Severe Combined Immunodeficiency Disease.
Blanco Elena et al. Frontiers in immunology 2020 11608653 - Current Clinical Applications of in vivo Gene Therapy with AAVs.
Mendell Jerry R et al. Molecular therapy : the journal of the American Society of Gene Therapy 2020 Dec - Gene therapy randomised clinical trials in Europe - a review paper of methodology and design.
Ilieva Krassimira et al. Journal of market access & health policy 2020 Nov 8(1) 1847808 - Post-Transcriptional Genetic Silencing of BCL11A to Treat Sickle Cell Disease.
Esrick Erica B et al. The New England journal of medicine 2020 Dec - Patient and public perspectives on cell and gene therapies: a systematic review.
Aiyegbusi Olalekan Lee et al. Nature communications 2020 11(1) 6265 - Gene therapy using haematopoietic stem and progenitor cells.
Ferrari Giuliana et al. Nature reviews. Genetics 2020 Dec - Cell and gene therapy: a snapshot of investor perspectives.
Moody Jennifer et al. Cytotherapy 2020 Dec - Assessing the current knowledge, attitude and behaviour of adolescents and young adults living with haemophilia.
Mohan Richa et al. Haemophilia : the official journal of the World Federation of Hemophilia 2020 Dec - Bilateral visual improvement with unilateral gene therapy injection for Leber hereditary optic neuropathy
P Yu et al, Sci Trans Med, December 9, 2020
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