Literature DB >> 25849425

A shot in the bone corrects a genetic disease.

Brian D Brown1.   

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Year:  2015        PMID： 25849425      PMCID： PMC4395793          DOI： 10.1038/mt.2015.38

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


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  11 in total

1.  Role of bone marrow transplantation for correcting hemophilia A in mice.

Authors:  Antonia Follenzi; Sanj Raut; Simone Merlin; Rita Sarkar; Sanjeev Gupta
Journal:  Blood       Date:  2012-02-24       Impact factor: 22.113

Review 2.  Gene therapy for hemophilia.

Authors:  M K Chuah; H Evens; T VandenDriessche
Journal:  J Thromb Haemost       Date:  2013-06       Impact factor: 5.824

3.  Sustained phenotypic correction of hemophilia a mice following oncoretroviral-mediated expression of a bioengineered human factor VIII gene in long-term hematopoietic repopulating cells.

Authors:  Morvarid Moayeri; Ali Ramezani; Richard A Morgan; Teresa S Hawley; Robert G Hawley
Journal:  Mol Ther       Date:  2004-11       Impact factor: 11.454

4.  Lentivirus-mediated platelet gene therapy of murine hemophilia A with pre-existing anti-factor VIII immunity.

Authors:  E L Kuether; J A Schroeder; S A Fahs; B C Cooley; Y Chen; R R Montgomery; D A Wilcox; Q Shi
Journal:  J Thromb Haemost       Date:  2012-08       Impact factor: 5.824

5.  Lentivirus-mediated platelet-derived factor VIII gene therapy in murine haemophilia A.

Authors:  Q Shi; D A Wilcox; S A Fahs; J Fang; B D Johnson; L M DU; D Desai; R R Montgomery
Journal:  J Thromb Haemost       Date:  2007-02       Impact factor: 5.824

6.  Intraosseous delivery of lentiviral vectors targeting factor VIII expression in platelets corrects murine hemophilia A.

Authors:  Xuefeng Wang; Simon C Shin; Andy F J Chiang; Iram Khan; Dao Pan; David J Rawlings; Carol H Miao
Journal:  Mol Ther       Date:  2015-02-06       Impact factor: 11.454

Review 7.  Ex vivo gene transfer and correction for cell-based therapies.

Authors:  Luigi Naldini
Journal:  Nat Rev Genet       Date:  2011-03-29       Impact factor: 53.242

8.  Gene therapy for immunodeficiency due to adenosine deaminase deficiency.

Authors:  Alessandro Aiuti; Federica Cattaneo; Stefania Galimberti; Ulrike Benninghoff; Barbara Cassani; Luciano Callegaro; Samantha Scaramuzza; Grazia Andolfi; Massimiliano Mirolo; Immacolata Brigida; Antonella Tabucchi; Filippo Carlucci; Martha Eibl; Memet Aker; Shimon Slavin; Hamoud Al-Mousa; Abdulaziz Al Ghonaium; Alina Ferster; Andrea Duppenthaler; Luigi Notarangelo; Uwe Wintergerst; Rebecca H Buckley; Marco Bregni; Sarah Marktel; Maria Grazia Valsecchi; Paolo Rossi; Fabio Ciceri; Roberto Miniero; Claudio Bordignon; Maria-Grazia Roncarolo
Journal:  N Engl J Med       Date:  2009-01-29       Impact factor: 91.245

9.  Rapamycin relieves lentiviral vector transduction resistance in human and mouse hematopoietic stem cells.

Authors:  Cathy X Wang; Blythe D Sather; Xuefeng Wang; Jennifer Adair; Iram Khan; Swati Singh; Shanshan Lang; Amie Adams; Gabrielle Curinga; Hans-Peter Kiem; Carol H Miao; David J Rawlings; Bruce E Torbett
Journal:  Blood       Date:  2014-06-09       Impact factor: 22.113

10.  Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy.

Authors:  Alessandra Biffi; Eugenio Montini; Laura Lorioli; Martina Cesani; Francesca Fumagalli; Tiziana Plati; Cristina Baldoli; Sabata Martino; Andrea Calabria; Sabrina Canale; Fabrizio Benedicenti; Giuliana Vallanti; Luca Biasco; Simone Leo; Nabil Kabbara; Gianluigi Zanetti; William B Rizzo; Nalini A L Mehta; Maria Pia Cicalese; Miriam Casiraghi; Jaap J Boelens; Ubaldo Del Carro; David J Dow; Manfred Schmidt; Andrea Assanelli; Victor Neduva; Clelia Di Serio; Elia Stupka; Jason Gardner; Christof von Kalle; Claudio Bordignon; Fabio Ciceri; Attilio Rovelli; Maria Grazia Roncarolo; Alessandro Aiuti; Maria Sessa; Luigi Naldini
Journal:  Science       Date:  2013-07-11       Impact factor: 47.728
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  3 in total

1.  Zebrafish embryonic stromal trunk (ZEST) cells support hematopoietic stem and progenitor cell (HSPC) proliferation, survival, and differentiation.

Authors:  Clyde Campbell; Tammy Su; Ryan P Lau; Arpit Shah; Payton C Laurie; Brenda Avalos; Julian Aggio; Elena Harris; David Traver; David L Stachura
Journal:  Exp Hematol       Date:  2015-09-21       Impact factor: 3.084

2.  Enhancing therapeutic efficacy of in vivo platelet-targeted gene therapy in hemophilia A mice.

Authors:  Xuefeng Wang; Richard Y Fu; Chong Li; Chun-Yu Chen; Jenni Firrman; Barbara A Konkle; Junping Zhang; Lei Li; Weidong Xiao; Mortimer Poncz; Carol H Miao
Journal:  Blood Adv       Date:  2020-11-24

Review 3.  Clinical development of gene therapy: results and lessons from recent successes.

Authors:  Sandeep Rp Kumar; David M Markusic; Moanaro Biswas; Katherine A High; Roland W Herzog
Journal:  Mol Ther Methods Clin Dev       Date:  2016-05-25       Impact factor: 6.698
  3 in total

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