Literature DB >> 21358702

Hemophilia gene therapy: a Holy Grail found.

Katherine P Ponder.   

Abstract

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Year:  2011        PMID： 21358702      PMCID： PMC3048193          DOI： 10.1038/mt.2011.13

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


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  21 in total

Review 1.  Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges.

Authors:  Federico Mingozzi; Katherine A High
Journal:  Nat Rev Genet       Date:  2011-05       Impact factor: 53.242

2.  AAV8-mediated hepatic gene transfer in infant rhesus monkeys (Macaca mulatta).

Authors:  Lili Wang; Peter Bell; Jianping Lin; Roberto Calcedo; Alice F Tarantal; James M Wilson
Journal:  Mol Ther       Date:  2011-08-02       Impact factor: 11.454

3.  A barrel of monkeys: scAAV8 gene therapy for hemophilia in nonhuman primates.

Authors:  Randy J Chandler; Charles P Venditti
Journal:  Mol Ther       Date:  2011-05       Impact factor: 11.454

4.  Impact of pre-existing immunity on gene transfer to nonhuman primate liver with adeno-associated virus 8 vectors.

Authors:  Lili Wang; Roberto Calcedo; Peter Bell; Jianping Lin; Rebecca L Grant; Don L Siegel; James M Wilson
Journal:  Hum Gene Ther       Date:  2011-06-08       Impact factor: 5.695

Review 5.  State-of-the-art gene-based therapies: the road ahead.

Authors:  Mark A Kay
Journal:  Nat Rev Genet       Date:  2011-04-06       Impact factor: 53.242

6.  Limitations of encapsidation of recombinant self-complementary adeno-associated viral genomes in different serotype capsids and their quantitation.

Authors:  Yuan Wang; Chen Ling; Liujiang Song; Lina Wang; George V Aslanidi; Mengqun Tan; Changquan Ling; Arun Srivastava
Journal:  Hum Gene Ther Methods       Date:  2012-08       Impact factor: 2.396

7.  Gene therapy for hemophilia: addressing the coming challenges of affordability and accessibility.

Authors:  Mark W Skinner
Journal:  Mol Ther       Date:  2013-01       Impact factor: 11.454

Review 8.  Gene therapy for haemophilia: prospects and challenges to prevent or reverse inhibitor formation.

Authors:  David W Scott; Jay N Lozier
Journal:  Br J Haematol       Date:  2011-11-07       Impact factor: 6.998

9.  Prolonged Expression of Secreted Enzymes in Dogs After Liver-Directed Delivery of Sleeping Beauty Transposons: Implications for Non-Viral Gene Therapy of Systemic Disease.

Authors:  Elena L Aronovich; Kendra A Hyland; Bryan C Hall; Jason B Bell; Erik R Olson; Myra Urness Rusten; David W Hunter; N Matthew Ellinwood; R Scott McIvor; Perry B Hackett
Journal:  Hum Gene Ther       Date:  2017-05-19       Impact factor: 5.695

10.  In vivo enrichment of genetically manipulated platelets corrects the murine hemophilic phenotype and induces immune tolerance even using a low multiplicity of infection.

Authors:  J A Schroeder; Y Chen; J Fang; D A Wilcox; Q Shi
Journal:  J Thromb Haemost       Date:  2014-07-17       Impact factor: 5.824
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