Literature DB >> 32396618

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

Matthew M Hsieh1, Melissa Bonner2, Francis John Pierciey2, Naoya Uchida1, James Rottman2, Laura Demopoulos2, Manfred Schmidt3, Julie Kanter4, Mark C Walters5, Alexis A Thompson6,7, Mohammed Asmal2, John F Tisdale1.   

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Year:  2020        PMID: 32396618      PMCID: PMC7218414          DOI: 10.1182/bloodadvances.2019001330

Source DB:  PubMed          Journal:  Blood Adv        ISSN: 2473-9529


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  30 in total

1.  Accelerated leukemic transformation after haplo-identical transplantation for hydroxyurea-treated sickle cell disease.

Authors:  Murali Janakiram; Amit Verma; Yanhua Wang; Anjali Budhathoki; Jaime Suarez Londono; Irina Murakhovskaya; Ira Braunschweig; Caterina P Minniti
Journal:  Leuk Lymphoma       Date:  2017-06-07

Review 2.  The incidence of secondary leukemias.

Authors:  G Leone; L Mele; A Pulsoni; F Equitani; L Pagano
Journal:  Haematologica       Date:  1999-10       Impact factor: 9.941

3.  Antibody Conditioning Enables MHC-Mismatched Hematopoietic Stem Cell Transplants and Organ Graft Tolerance.

Authors:  Benson M George; Kevin S Kao; Hye-Sook Kwon; Brenda J Velasco; Jessica Poyser; Angela Chen; Alan C Le; Akanksha Chhabra; Cassandra E Burnett; Devon Cajuste; Malachia Hoover; Kyle M Loh; Judith A Shizuru; Irving L Weissman
Journal:  Cell Stem Cell       Date:  2019-06-13       Impact factor: 24.633

Review 4.  Gene therapy targeting haematopoietic stem cells for inherited diseases: progress and challenges.

Authors:  Marina Cavazzana; Frederic D Bushman; Annarita Miccio; Isabelle André-Schmutz; Emmanuelle Six
Journal:  Nat Rev Drug Discov       Date:  2019-06       Impact factor: 84.694

5.  Stable mixed hematopoietic chimerism after bone marrow transplantation for sickle cell anemia.

Authors:  M C Walters; M Patience; W Leisenring; Z R Rogers; V M Aquino; G R Buchanan; I A Roberts; A M Yeager; L Hsu; T Adamkiewicz; J Kurtzberg; E Vichinsky; B Storer; R Storb; K M Sullivan
Journal:  Biol Blood Marrow Transplant       Date:  2001       Impact factor: 5.742

Review 6.  Systematic review: Hydroxyurea for the treatment of adults with sickle cell disease.

Authors:  Sophie Lanzkron; John J Strouse; Renee Wilson; Mary Catherine Beach; Carlton Haywood; HaeSong Park; Catherine Witkop; Eric B Bass; Jodi B Segal
Journal:  Ann Intern Med       Date:  2008-05-05       Impact factor: 25.391

Review 7.  Treating Immunodeficiency through HSC Gene Therapy.

Authors:  Claire Booth; H Bobby Gaspar; Adrian J Thrasher
Journal:  Trends Mol Med       Date:  2016-03-15       Impact factor: 11.951

8.  Hematopoietic stem cell transplantation in immunocompetent hosts without radiation or chemotherapy.

Authors:  Akanksha Chhabra; Aaron M Ring; Kipp Weiskopf; Peter John Schnorr; Sydney Gordon; Alan C Le; Hye-Sook Kwon; Nan Guo Ring; Jens Volkmer; Po Yi Ho; Serena Tseng; Irving L Weissman; Judith A Shizuru
Journal:  Sci Transl Med       Date:  2016-08-10       Impact factor: 17.956

9.  Selective hematopoietic stem cell ablation using CD117-antibody-drug-conjugates enables safe and effective transplantation with immunity preservation.

Authors:  Agnieszka Czechowicz; Rahul Palchaudhuri; Amelia Scheck; Yu Hu; Jonathan Hoggatt; Borja Saez; Wendy W Pang; Michael K Mansour; Tiffany A Tate; Yan Yi Chan; Emily Walck; Gerlinde Wernig; Judith A Shizuru; Florian Winau; David T Scadden; Derrick J Rossi
Journal:  Nat Commun       Date:  2019-02-06       Impact factor: 14.919

10.  Karyotypic and fluorescent in situ hybridization study of the centromere of chromosome 7 in secondary myeloid neoplasms.

Authors:  Roberta Sandra da Silva Tanizawa; Cristina Aiko Kumeda; Raymundo Soares de Azevedo Neto; Aline de Medeiros Leal; Patrícia de Barros Ferreira; Elvira Deolinda Rodrigues Pereira Velloso
Journal:  Rev Bras Hematol Hemoter       Date:  2011
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  23 in total

Review 1.  Fetal hemoglobin in sickle cell anemia.

Authors:  Martin H Steinberg
Journal:  Blood       Date:  2020-11-19       Impact factor: 22.113

2.  Post-Transcriptional Genetic Silencing of BCL11A to Treat Sickle Cell Disease.

Authors:  Erica B Esrick; Leslie E Lehmann; Alessandra Biffi; Maureen Achebe; Christian Brendel; Marioara F Ciuculescu; Heather Daley; Brenda MacKinnon; Emily Morris; Amy Federico; Daniela Abriss; Kari Boardman; Radia Khelladi; Kit Shaw; Helene Negre; Olivier Negre; Sarah Nikiforow; Jerome Ritz; Sung-Yun Pai; Wendy B London; Colleen Dansereau; Matthew M Heeney; Myriam Armant; John P Manis; David A Williams
Journal:  N Engl J Med       Date:  2020-12-05       Impact factor: 91.245

Review 3.  β-Thalassemia: evolving treatment options beyond transfusion and iron chelation.

Authors:  Arielle L Langer; Erica B Esrick
Journal:  Hematology Am Soc Hematol Educ Program       Date:  2021-12-10

Review 4.  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 5.  The transformative potential of HSC gene therapy as a genetic medicine.

Authors:  Pervinder Sagoo; H Bobby Gaspar
Journal:  Gene Ther       Date:  2021-05-26       Impact factor: 5.250

6.  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

Review 7.  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 8.  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 9.  Innovative Treatments for Rare Anemias.

Authors:  Maria Domenica Cappellini; Alessia Marcon; Bruno Fattizzo; Irene Motta
Journal:  Hemasphere       Date:  2021-06-01

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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