Literature DB >> 31578501

First gene therapy for β-thalassemia approved.

Charlotte Harrison.   

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Year:  2019        PMID： 31578501     DOI： 10.1038/d41587-019-00026-3

Source DB:  PubMed          Journal:  Nat Biotechnol        ISSN： 1087-0156            Impact factor:   54.908


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  9 in total

Review 1.  Emerging technologies for cystic fibrosis transmembrane conductance regulator restoration in all people with CF.

Authors:  Marie E Egan
Journal:  Pediatr Pulmonol       Date:  2021-02

2.  Health State Utilities for Sickle Cell Disease: A Catalog Prepared From a Systematic Review.

Authors:  Boshen Jiao; Anirban Basu; Scott Ramsey; Joshua Roth; M A Bender; Dalyna Quach; Beth Devine
Journal:  Value Health       Date:  2021-09-04       Impact factor: 5.725

3.  Peptide nucleic acid-dependent artifact can lead to false-positive triplex gene editing signals.

Authors:  Pui Yan Ho; Zhen Zhang; Mark E Hayes; Andrew Curd; Carla Dib; Maire Rayburn; Sze Nok Tam; Tumul Srivastava; Brandon Hriniak; Xiao-Jun Li; Scott Leonard; Lan Wang; Somayeh Tarighat; Derek S Sim; Mark Fiandaca; James M Coull; Allen Ebens; Marshall Fordyce; Agnieszka Czechowicz
Journal:  Proc Natl Acad Sci U S A       Date:  2021-11-09       Impact factor: 11.205

Review 4.  Treatment strategies for glucose-6-phosphate dehydrogenase deficiency: past and future perspectives.

Authors:  Adriana A Garcia; Ana Koperniku; Julio C B Ferreira; Daria Mochly-Rosen
Journal:  Trends Pharmacol Sci       Date:  2021-08-10       Impact factor: 17.638

Review 5.  The Use of Cost-Effectiveness Analysis in Sickle Cell Disease: A Critical Review of the Literature.

Authors:  Boshen Jiao; Anirban Basu; Joshua Roth; M Bender; Ilsa Rovira; Traci Clemons; Dalyna Quach; Scott Ramsey; Beth Devine
Journal:  Pharmacoeconomics       Date:  2021-08-09       Impact factor: 4.981

Review 6.  On the Corner of Models and Cure: Gene Editing in Cystic Fibrosis.

Authors:  Marjolein Ensinck; Angélique Mottais; Claire Detry; Teresinha Leal; Marianne S Carlon
Journal:  Front Pharmacol       Date:  2021-04-27       Impact factor: 5.810

7.  Development of a conceptual model for evaluating new non-curative and curative therapies for sickle cell disease.

Authors:  Kate M Johnson; Boshen Jiao; M A Bender; Scott D Ramsey; Beth Devine; Anirban Basu
Journal:  PLoS One       Date:  2022-04-28       Impact factor: 3.752

8.  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

Review 9.  Effect of Glucocorticosteroids in Diamond-Blackfan Anaemia: Maybe Not as Elusive as It Seems.

Authors:  Zuzana Macečková; Agáta Kubíčková; Juan Bautista De Sanctis; Marian Hajdúch
Journal:  Int J Mol Sci       Date:  2022-02-08       Impact factor: 5.923
  9 in total

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