Potent and uniform fetal hemoglobin induction via base editing-Reference-Cited by-全球学者库

Potent and uniform fetal hemoglobin induction via base editing

Published:2023-07 Issue:7 Volume:55 Page:1210-1220
ISSN:1061-4036
Container-title:Nature Genetics
language:en
Short-container-title:Nat Genet

Author:

Mayuranathan Thiyagaraj^ORCID,Newby Gregory A.^ORCID,Feng Ruopeng,Yao Yu,Mayberry Kalin D.,Lazzarotto Cicera R.^ORCID,Li Yichao^ORCID,Levine Rachel M.^ORCID,Nimmagadda Nikitha^ORCID,Dempsey Erin^ORCID,Kang Guolian^ORCID,Porter Shaina N.,Doerfler Phillip A.^ORCID,Zhang Jingjing,Jang Yoonjeong,Chen Jingjing^ORCID,Bell Henry W.^ORCID,Crossley Merlin^ORCID,Bhoopalan Senthil Velan^ORCID,Sharma Akshay^ORCID,Tisdale John F.,Pruett-Miller Shondra M.^ORCID,Cheng Yong^ORCID,Tsai Shengdar Q.^ORCID,Liu David R.^ORCID,Weiss Mitchell J.^ORCID,Yen Jonathan S.^ORCID

Funder

U.S. Department of Health & Human Services | NIH | National Heart, Lung, and Blood Institute

St. Jude Children’s Research Hospital

American Lebanese Syrian Associated Charities

Assisi Foundation of Memphis

Bill and Melinda Gates Foundation

Helen Hay Whitney Foundation

Cooley’s Anemia Foundation

U.S. Department of Health & Human Services | NIH | National Institute of Diabetes and Digestive and Kidney Diseases

American Society of Hematology

Doris Duke Charitable Foundation

U.S. Department of Health & Human Services | NIH | National Institute of General Medical Sciences

U.S. Department of Health & Human Services | NIH | National Institute of Allergy and Infectious Diseases

U.S. Department of Health & Human Services | NIH | National Human Genome Research Institute

U.S. Department of Health & Human Services | NIH | National Institute of Biomedical Imaging and Bioengineering

Howard Hughes Medical Institute

Publisher

Springer Science and Business Media LLC

Subject

Genetics

Link

https://www.nature.com/articles/s41588-023-01434-7.pdf

Reference70 articles.

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2. Taher, A. T., Musallam, K. M. & Cappellini, M. D. Beta-thalassemias. N. Engl. J. Med. 384, 727–743 (2021).

3. Doerfler, P. A. et al. Genetic therapies for the first molecular disease. J. Clin. Invest. 131, e146394 (2021).

4. Esrick, E. B. et al. Post-transcriptional genetic silencing of BCL11A to treat sickle cell disease. N. Engl. J. Med. 384, 205–215 (2021).

5. Frangoul, H. et al. CRISPR-Cas9 gene editing for sickle cell disease and β-thalassemia. N. Engl. J. Med. 384, 252–260 (2021).

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