Literature DB >> 33743192

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

Alexis Leonard1, John F Tisdale2.   

Abstract

Entities:  

Mesh：

Year:  2021        PMID： 33743192      PMCID： PMC8058500          DOI： 10.1016/j.ymthe.2021.03.010

Source DB:  PubMed          Journal:  Mol Ther        ISSN： 1525-0016            Impact factor:   11.454


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  8 in total

1.  Clonal Hematopoiesis Associated With Adverse Outcomes After Autologous Stem-Cell Transplantation for Lymphoma.

Authors:  Christopher J Gibson; R Coleman Lindsley; Vatche Tchekmedyian; Brenton G Mar; Jiantao Shi; Siddhartha Jaiswal; Alysia Bosworth; Liton Francisco; Jianbo He; Anita Bansal; Elizabeth A Morgan; Ann S Lacasce; Arnold S Freedman; David C Fisher; Eric Jacobsen; Philippe Armand; Edwin P Alyea; John Koreth; Vincent Ho; Robert J Soiffer; Joseph H Antin; Jerome Ritz; Sarah Nikiforow; Stephen J Forman; Franziska Michor; Donna Neuberg; Ravi Bhatia; Smita Bhatia; Benjamin L Ebert
Journal:  J Clin Oncol       Date:  2017-01-09       Impact factor: 44.544

2.  Increased risk of leukemia among sickle cell disease patients in California.

Authors:  Ann Brunson; Theresa H M Keegan; Heejung Bang; Anjlee Mahajan; Susan Paulukonis; Ted Wun
Journal:  Blood       Date:  2017-08-22       Impact factor: 22.113

Review 3.  Therapy-related myeloid neoplasms: when genetics and environment collide.

Authors:  Megan E McNerney; Lucy A Godley; Michelle M Le Beau
Journal:  Nat Rev Cancer       Date:  2017-08-24       Impact factor: 60.716

4.  Baseline TP53 mutations in adults with SCD developing myeloid malignancy following hematopoietic cell transplantation.

Authors:  Jack Y Ghannam; Xin Xu; Irina Maric; Laura Dillon; Yuesheng Li; Matthew M Hsieh; Christopher S Hourigan; Courtney D Fitzhugh
Journal:  Blood       Date:  2020-04-02       Impact factor: 22.113

5.  Myelodysplastic syndrome unrelated to lentiviral vector in a patient treated with gene therapy for sickle cell disease.

Authors:  Matthew M Hsieh; Melissa Bonner; Francis John Pierciey; Naoya Uchida; James Rottman; Laura Demopoulos; Manfred Schmidt; Julie Kanter; Mark C Walters; Alexis A Thompson; Mohammed Asmal; John F Tisdale
Journal:  Blood Adv       Date:  2020-05-12

6.  Myeloid neoplasms in the setting of sickle cell disease: an intrinsic association with the underlying condition rather than a coincidence; report of 4 cases and review of the literature.

Authors:  Yang Li; Jake Maule; Jadee L Neff; Chad M McCall; Sarah Rapisardo; Anand S Lagoo; Lian-He Yang; Regina D Crawford; Yue Zhao; Endi Wang
Journal:  Mod Pathol       Date:  2019-08-01       Impact factor: 7.842

7.  Effect of donor type and conditioning regimen intensity on allogeneic transplantation outcomes in patients with sickle cell disease: a retrospective multicentre, cohort study.

Authors:  Mary Eapen; Ruta Brazauskas; Mark C Walters; Françoise Bernaudin; Khalid Bo-Subait; Courtney D Fitzhugh; Jane S Hankins; Julie Kanter; Joerg J Meerpohl; Javier Bolaños-Meade; Julie A Panepinto; Damiano Rondelli; Shalini Shenoy; Joi Williamson; Teonna L Woolford; Eliane Gluckman; John E Wagner; John F Tisdale
Journal:  Lancet Haematol       Date:  2019-09-05       Impact factor: 18.959
  8 in total
  12 in total

1.  Gene therapy for sickle cell disease: where we are now?

Authors:  Julie Kanter; Corey Falcon
Journal:  Hematology Am Soc Hematol Educ Program       Date:  2021-12-10

Review 2.  Editing outside the body: Ex vivo gene-modification for β-hemoglobinopathy cellular therapy.

Authors:  Tolulope O Rosanwo; Daniel E Bauer
Journal:  Mol Ther       Date:  2021-10-08       Impact factor: 11.454

Review 3.  Microfluidic methods to advance mechanistic understanding and translational research in sickle cell disease.

Authors:  Melissa Azul; Eudorah F Vital; Wilbur A Lam; David K Wood; Joan D Beckman
Journal:  Transl Res       Date:  2022-03-27       Impact factor: 10.171

Review 4.  Hematopoietic Stem Cell Gene-Addition/Editing Therapy in Sickle Cell Disease.

Authors:  Paula Germino-Watnick; Malikiya Hinds; Anh Le; Rebecca Chu; Xiong Liu; Naoya Uchida
Journal:  Cells       Date:  2022-06-04       Impact factor: 7.666

Review 5.  Genetic therapies for the first molecular disease.

Authors:  Phillip A Doerfler; Akshay Sharma; Jerlym S Porter; Yan Zheng; John F Tisdale; Mitchell J Weiss
Journal:  J Clin Invest       Date:  2021-04-15       Impact factor: 14.808

Review 6.  Understanding and overcoming adverse consequences of genome editing on hematopoietic stem and progenitor cells.

Authors:  Byung-Chul Lee; Richard J Lozano; Cynthia E Dunbar
Journal:  Mol Ther       Date:  2021-09-10       Impact factor: 11.454

Review 7.  Gene therapy for sickle cell disease: moving from the bench to the bedside.

Authors:  Allistair A Abraham; John F Tisdale
Journal:  Blood       Date:  2021-09-16       Impact factor: 25.476

8.  Old versus new gene therapy for globin disorders.

Authors:  Roland W Herzog; Jörg Bungert
Journal:  Mol Ther       Date:  2021-05-07       Impact factor: 12.910

9.  3' UTR-truncated HMGA2 overexpression induces non-malignant in vivo expansion of hematopoietic stem cells in non-human primates.

Authors:  Melissa A Bonner; Antonio Morales-Hernández; Sheng Zhou; Zhijun Ma; Jose Condori; Yong-Dong Wang; Soghra Fatima; Lance E Palmer; Laura J Janke; Stephanie Fowler; Brian P Sorrentino; Shannon McKinney-Freeman
Journal:  Mol Ther Methods Clin Dev       Date:  2021-05-01       Impact factor: 6.698

Review 10.  Research in Sickle Cell Disease: From Bedside to Bench to Bedside.

Authors:  Gabriel Salinas Cisneros; Swee Lay Thein
Journal:  Hemasphere       Date:  2021-06-01
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