Development and IND-enabling studies of a novel Cas9 genome-edited autologous CD34+ cell therapy to induce fetal hemoglobin for sickle cell disease

Author:

Katta Varun,O’Keefe Kiera,Li Yichao,Mayuranathan Thiyagaraj,Lazzarotto Cicera R.,Wood Rachael K.,Levine Rachel M.,Powers Alicia,Mayberry Kalin,Manquen Garret,Yao Yu,Zhang Jingjing,Jang Yoonjeong,Nimmagadda Nikitha,Dempsey Erin A.,Lee GaHyun,Uchida Naoya,Cheng Yong,Fazio Frank,Lockey Tim,Meagher Mike,Sharma Akshay,Tisdale John F.,Zhou Sheng,Yen Jonathan S.,Weiss Mitchell J.,Tsai Shengdar Q.ORCID

Funder

St Jude Children's Research Hospital

Doris Duke Charitable Foundation

NIH

Publisher

Elsevier BV

Reference79 articles.

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2. A specific chemical difference between the globins of normal human and sickle-cell anaemia haemoglobin;Ingram;Nature,1956

3. Pathogenesis and treatment of sickle cell disease;Bunn;N. Engl. J. Med.,1997

4. Sickle cell disease;Kato;Nat. Rev. Dis. Primers,2018

5. Recent advances in sickle-cell disease therapies: A review of voxelotor, crizanlizumab, and L-glutamine;Migotsky;Pharmacy (Basel),2022

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