Literature DB >> 24316783

Gene therapy: the promise of a permanent cure.

Christopher D Porada1, Christopher Stem, Graca Almeida-Porada.   

Abstract

Gene therapy offers the possibility of a permanent cure for any of the more than 10,000 human diseases caused by a defect in a single gene. Among these diseases, the hemophilias represent an ideal target, and studies in both animals and humans have provided evidence that a permanent cure for hemophilia is within reach.

Entities:  

Mesh：

Year:  2013        PMID： 24316783      PMCID： PMC8092353     

Source DB:  PubMed          Journal:  N C Med J        ISSN： 0029-2559


  43 in total

Review 1.  In utero hematopoietic stem cell transplantation: ontogenic opportunities and biologic barriers.

Authors:  A W Flake; E D Zanjani
Journal:  Blood       Date:  1999-10-01       Impact factor: 22.113

2.  Induction of stable prenatal tolerance to beta-galactosidase by in utero gene transfer into preimmune sheep fetuses.

Authors:  N D Tran; C D Porada; G Almeida-Porada; H A Glimp; W F Anderson; E D Zanjani
Journal:  Blood       Date:  2001-06-01       Impact factor: 22.113

3.  Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B.

Authors:  Valder R Arruda; Hansell H Stedman; Virginia Haurigot; George Buchlis; Stefano Baila; Patricia Favaro; Yifeng Chen; Helen G Franck; Shangzhen Zhou; J Fraser Wright; Linda B Couto; Haiyan Jiang; Glenn F Pierce; Dwight A Bellinger; Federico Mingozzi; Timothy C Nichols; Katherine A High
Journal:  Blood       Date:  2010-03-24       Impact factor: 22.113

4.  Male germ-line cells are at risk following direct-injection retroviral-mediated gene transfer in utero.

Authors:  Christopher D Porada; Paul J Park; Joe Tellez; Ferhat Ozturk; Hudson A Glimp; Graça Almeida-Porada; Esmail D Zanjani
Journal:  Mol Ther       Date:  2005-10       Impact factor: 11.454

5.  Long-term correction of inhibitor-prone hemophilia B dogs treated with liver-directed AAV2-mediated factor IX gene therapy.

Authors:  Glenn P Niemeyer; Roland W Herzog; Jane Mount; Valder R Arruda; D Michael Tillson; John Hathcock; Frederik W van Ginkel; Katherine A High; Clinton D Lothrop
Journal:  Blood       Date:  2008-10-28       Impact factor: 22.113

Review 6.  The gene therapy journey for hemophilia: are we there yet?

Authors:  Katherine A High
Journal:  Hematology Am Soc Hematol Educ Program       Date:  2012

Review 7.  Animal models of hemophilia.

Authors:  Denise E Sabatino; Timothy C Nichols; Elizabeth Merricks; Dwight A Bellinger; Roland W Herzog; Paul E Monahan
Journal:  Prog Mol Biol Transl Sci       Date:  2012       Impact factor: 3.622

8.  Efficacy and safety of long-term prophylaxis in severe hemophilia A dogs following liver gene therapy using AAV vectors.

Authors:  Denise E Sabatino; Amy M Lange; Ekaterina S Altynova; Rita Sarkar; Shangzhen Zhou; Elizabeth P Merricks; Helen G Franck; Timothy C Nichols; Valder R Arruda; Haig H Kazazian
Journal:  Mol Ther       Date:  2010-11-16       Impact factor: 11.454

Review 9.  Gene therapy for haemophilia: a long and winding road.

Authors:  K A High
Journal:  J Thromb Haemost       Date:  2011-07       Impact factor: 5.824

10.  In utero gene therapy: transfer and long-term expression of the bacterial neo(r) gene in sheep after direct injection of retroviral vectors into preimmune fetuses.

Authors:  C D Porada; N Tran; M Eglitis; R C Moen; L Troutman; A W Flake; Y Zhao; W F Anderson; E D Zanjani
Journal:  Hum Gene Ther       Date:  1998-07-20       Impact factor: 5.695
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  5 in total

1.  Investigating Optimal Autologous Cellular Platforms for Prenatal or Perinatal Factor VIII Delivery to Treat Hemophilia A.

Authors:  Christopher Stem; Christopher Rodman; Ritu M Ramamurthy; Sunil George; Diane Meares; Andrew Farland; Anthony Atala; Christopher B Doering; H Trent Spencer; Christopher D Porada; Graça Almeida-Porada
Journal:  Front Cell Dev Biol       Date:  2021-08-10

2.  Generation and characterization of transgenic mouse mesenchymal stem cell lines expressing hIGF-1 or hG-CSF.

Authors:  Gabrielle V M Gonçalves; Daniela N Silva; Rejane H Carvalho; Bruno S F Souza; Kátia Nunes da Silva; Juliana F Vasconcelos; Bruno D Paredes; Carolina K V Nonaka; Ricardo Ribeiro-Dos-Santos; Milena B P Soares
Journal:  Cytotechnology       Date:  2017-09-02       Impact factor: 2.058

3.  Mechanistic Insights into Factor VIII Immune Tolerance Induction via Prenatal Cell Therapy in Hemophilia A.

Authors:  Martin Rodriguez; Christopher D Porada; Graҫa Almeida-Porada
Journal:  Curr Stem Cell Rep       Date:  2019-11-20

Review 4.  In utero stem cell transplantation and gene therapy: rationale, history, and recent advances toward clinical application.

Authors:  Graça Almeida-Porada; Anthony Atala; Christopher D Porada
Journal:  Mol Ther Methods Clin Dev       Date:  2016-03-30       Impact factor: 6.698

Review 5.  Induced Pluripotent Stem Cells (iPSCs) and Gene Therapy: A New Era for the Treatment of Neurological Diseases.

Authors:  Giulia Paolini Sguazzi; Valentina Muto; Marco Tartaglia; Enrico Bertini; Claudia Compagnucci
Journal:  Int J Mol Sci       Date:  2021-12-20       Impact factor: 5.923
  5 in total

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