Literature DB >> 33961803

Old versus new gene therapy for globin disorders.

Roland W Herzog1, Jörg Bungert2.   

Abstract

Entities:  

Mesh:

Substances:

Year:  2021        PMID: 33961803      PMCID: PMC8178449          DOI: 10.1016/j.ymthe.2021.04.031

Source DB:  PubMed          Journal:  Mol Ther        ISSN: 1525-0016            Impact factor:   12.910


× No keyword cloud information.
  4 in total

1.  CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia.

Authors:  Haydar Frangoul; David Altshuler; M Domenica Cappellini; Yi-Shan Chen; Jennifer Domm; Brenda K Eustace; Juergen Foell; Josu de la Fuente; Stephan Grupp; Rupert Handgretinger; Tony W Ho; Antonis Kattamis; Andrew Kernytsky; Julie Lekstrom-Himes; Amanda M Li; Franco Locatelli; Markus Y Mapara; Mariane de Montalembert; Damiano Rondelli; Akshay Sharma; Sujit Sheth; Sandeep Soni; Martin H Steinberg; Donna Wall; Angela Yen; Selim Corbacioglu
Journal:  N Engl J Med       Date:  2020-12-05       Impact factor: 91.245

2.  A pause in gene therapy: Reflecting on the unique challenges of sickle cell disease.

Authors:  Alexis Leonard; John F Tisdale
Journal:  Mol Ther       Date:  2021-03-19       Impact factor: 11.454

3.  Lentiviral vector ALS20 yields high hemoglobin levels with low genomic integrations for treatment of beta-globinopathies.

Authors:  Laura Breda; Valentina Ghiaccio; Naoto Tanaka; Danuta Jarocha; Yasuhiro Ikawa; Osheiza Abdulmalik; Alisa Dong; Carla Casu; Tobias D Raabe; Xiaochuan Shan; Gwenn A Danet-Desnoyers; Aoife M Doto; John Everett; Frederic D Bushman; Enrico Radaelli; Charles A Assenmacher; James C Tarrant; Natalie Hoepp; Ryo Kurita; Yukio Nakamura; Virginia Guzikowski; Kim Smith-Whitley; Janet L Kwiatkowski; Stefano Rivella
Journal:  Mol Ther       Date:  2021-01-29       Impact factor: 11.454

4.  Coordinated β-globin expression and α2-globin reduction in a multiplex lentiviral gene therapy vector for β-thalassemia.

Authors:  Tiwaporn Nualkaew; Karine Sii-Felice; Marie Giorgi; Bradley McColl; Julie Gouzil; Astrid Glaser; Hsiao P J Voon; Hsin Y Tee; George Grigoriadis; Saovaros Svasti; Suthat Fucharoen; Suradej Hongeng; Philippe Leboulch; Emmanuel Payen; Jim Vadolas
Journal:  Mol Ther       Date:  2021-05-01       Impact factor: 12.910

  4 in total

北京卡尤迪生物科技股份有限公司 © 2022-2023.