Literature DB >> 23517518

Gene therapy for rare diseases: summary of a National Institutes of Health workshop, September 13, 2012.

Marina O'Reilly1, Donald B Kohn, Jeffrey Bartlett, Janet Benson, Philip J Brooks, Barry J Byrne, Carlos Camozzi, Kenneth Cornetta, Ronald G Crystal, Yuman Fong, Linda Gargiulo, Rashmi Gopal-Srivastava, Katherine A High, Samuel G Jacobson, Robert C Jambou, Maureen Montgomery, Eugene Rosenthal, R Jude Samulski, Sonia I Skarlatos, Brian Sorrentino, James M Wilson, Yun Xie, Jacqueline Corrigan-Curay.   

Abstract

Gene therapy has shown clinical efficacy for several rare diseases, using different approaches and vectors. The Gene Therapy for Rare Diseases workshop, sponsored by the National Institutes of Health (NIH) Office of Biotechnology Activities and Office of Rare Diseases Research, brought together investigators from different disciplines to discuss the challenges and opportunities for advancing the field including means for enhancing data sharing for preclinical and clinical studies, development and utilization of available NIH resources, and interactions with the U.S. Food and Drug Administration.

Mesh：

Year:  2013        PMID： 23517518      PMCID： PMC3631014          DOI： 10.1089/hum.2013.064

Source DB:  PubMed          Journal:  Hum Gene Ther        ISSN： 1043-0342            Impact factor:   5.695


  16 in total

1.  Intramuscular administration of AAV1-lipoprotein lipase S447X lowers triglycerides in lipoprotein lipase-deficient patients.

Authors:  Erik S Stroes; Melchior C Nierman; Janneke J Meulenberg; Remco Franssen; Jaap Twisk; C Pieter Henny; Mario M Maas; Aeilko H Zwinderman; Colin Ross; Eleonora Aronica; Katherine A High; Marcel M Levi; Michael R Hayden; John J Kastelein; Jan Albert Kuivenhoven
Journal:  Arterioscler Thromb Vasc Biol       Date:  2008-09-18       Impact factor: 8.311

Review 2.  Leber congenital amaurosis due to RPE65 mutations and its treatment with gene therapy.

Authors:  Artur V Cideciyan
Journal:  Prog Retin Eye Res       Date:  2010-04-24       Impact factor: 21.198

3.  Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia.

Authors:  Marina Cavazzana-Calvo; Emmanuel Payen; Olivier Negre; Gary Wang; Kathleen Hehir; Floriane Fusil; Julian Down; Maria Denaro; Troy Brady; Karen Westerman; Resy Cavallesco; Beatrix Gillet-Legrand; Laure Caccavelli; Riccardo Sgarra; Leila Maouche-Chrétien; Françoise Bernaudin; Robert Girot; Ronald Dorazio; Geert-Jan Mulder; Axel Polack; Arthur Bank; Jean Soulier; Jérôme Larghero; Nabil Kabbara; Bruno Dalle; Bernard Gourmel; Gérard Socie; Stany Chrétien; Nathalie Cartier; Patrick Aubourg; Alain Fischer; Kenneth Cornetta; Frédéric Galacteros; Yves Beuzard; Eliane Gluckman; Frederick Bushman; Salima Hacein-Bey-Abina; Philippe Leboulch
Journal:  Nature       Date:  2010-09-16       Impact factor: 49.962

4.  Stem-cell gene therapy for the Wiskott-Aldrich syndrome.

Authors:  Kaan Boztug; Manfred Schmidt; Adrian Schwarzer; Pinaki P Banerjee; Inés Avedillo Díez; Ricardo A Dewey; Marie Böhm; Ali Nowrouzi; Claudia R Ball; Hanno Glimm; Sonja Naundorf; Klaus Kühlcke; Rainer Blasczyk; Irina Kondratenko; László Maródi; Jordan S Orange; Christof von Kalle; Christoph Klein
Journal:  N Engl J Med       Date:  2010-11-11       Impact factor: 91.245

5.  Efficacy of gene therapy for X-linked severe combined immunodeficiency.

Authors:  Salima Hacein-Bey-Abina; Julia Hauer; Annick Lim; Capucine Picard; Gary P Wang; Charles C Berry; Chantal Martinache; Frédéric Rieux-Laucat; Sylvain Latour; Bernd H Belohradsky; Lily Leiva; Ricardo Sorensen; Marianne Debré; Jean Laurent Casanova; Stephane Blanche; Anne Durandy; Frederic D Bushman; Alain Fischer; Marina Cavazzana-Calvo
Journal:  N Engl J Med       Date:  2010-07-22       Impact factor: 91.245

6.  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

7.  LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1.

Authors:  S Hacein-Bey-Abina; C Von Kalle; M Schmidt; M P McCormack; N Wulffraat; P Leboulch; A Lim; C S Osborne; R Pawliuk; E Morillon; R Sorensen; A Forster; P Fraser; J I Cohen; G de Saint Basile; I Alexander; U Wintergerst; T Frebourg; A Aurias; D Stoppa-Lyonnet; S Romana; I Radford-Weiss; F Gross; F Valensi; E Delabesse; E Macintyre; F Sigaux; J Soulier; L E Leiva; M Wissler; C Prinz; T H Rabbitts; F Le Deist; A Fischer; M Cavazzana-Calvo
Journal:  Science       Date:  2003-10-17       Impact factor: 47.728

8.  Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease.

Authors:  Stefan Stein; Marion G Ott; Stephan Schultze-Strasser; Anna Jauch; Barbara Burwinkel; Andrea Kinner; Manfred Schmidt; Alwin Krämer; Joachim Schwäble; Hanno Glimm; Ulrike Koehl; Carolin Preiss; Claudia Ball; Hans Martin; Gudrun Göhring; Kerstin Schwarzwaelder; Wolf-Karsten Hofmann; Kadin Karakaya; Sandrine Tchatchou; Rongxi Yang; Petra Reinecke; Klaus Kühlcke; Brigitte Schlegelberger; Adrian J Thrasher; Dieter Hoelzer; Reinhard Seger; Christof von Kalle; Manuel Grez
Journal:  Nat Med       Date:  2010-01-24       Impact factor: 53.440

9.  Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy.

Authors:  Nathalie Cartier; Salima Hacein-Bey-Abina; Cynthia C Bartholomae; Gabor Veres; Manfred Schmidt; Ina Kutschera; Michel Vidaud; Ulrich Abel; Liliane Dal-Cortivo; Laure Caccavelli; Nizar Mahlaoui; Véronique Kiermer; Denice Mittelstaedt; Céline Bellesme; Najiba Lahlou; François Lefrère; Stéphane Blanche; Muriel Audit; Emmanuel Payen; Philippe Leboulch; Bruno l'Homme; Pierre Bougnères; Christof Von Kalle; Alain Fischer; Marina Cavazzana-Calvo; Patrick Aubourg
Journal:  Science       Date:  2009-11-06       Impact factor: 47.728

10.  CD8(+) T-cell responses to adeno-associated virus capsid in humans.

Authors:  Federico Mingozzi; Marcela V Maus; Daniel J Hui; Denise E Sabatino; Samuel L Murphy; John E J Rasko; Margaret V Ragni; Catherine S Manno; Jurg Sommer; Haiyan Jiang; Glenn F Pierce; Hildegund C J Ertl; Katherine A High
Journal:  Nat Med       Date:  2007-03-18       Impact factor: 53.440
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  12 in total

Review 1.  What Is Next for Retinal Gene Therapy?

Authors:  Luk H Vandenberghe
Journal:  Cold Spring Harb Perspect Med       Date:  2015-04-15       Impact factor: 6.915

Review 2.  Development of gene therapy for blood disorders: an update.

Authors:  Arthur W Nienhuis
Journal:  Blood       Date:  2013-07-10       Impact factor: 22.113

3.  Pathway for approval of a gene therapy orphan product: treading new ground.

Authors:  Barry J Byrne
Journal:  Mol Ther       Date:  2013-08       Impact factor: 11.454

4.  Non-myeloablative conditioning with busulfan before hematopoietic stem cell transplantation leads to phenotypic correction of murine Bernard-Soulier syndrome.

Authors:  S Kanaji; S A Fahs; J Ware; R R Montgomery; Q Shi
Journal:  J Thromb Haemost       Date:  2014-08-26       Impact factor: 5.824

Review 5.  Promising and delivering gene therapies for vision loss.

Authors:  Livia S Carvalho; Luk H Vandenberghe
Journal:  Vision Res       Date:  2014-08-02       Impact factor: 1.886

Review 6.  Parkinson's disease gene therapy: success by design meets failure by efficacy.

Authors:  Raymond T Bartus; Marc S Weinberg; R Jude Samulski
Journal:  Mol Ther       Date:  2013-12-20       Impact factor: 11.454

7.  Developmental stage determines efficiency of gene transfer to muscle satellite cells by in utero delivery of adeno-associated virus vector serotype 2/9.

Authors:  David H Stitelman; Tim Brazelton; Archana Bora; Jeremy Traas; Demetri Merianos; Maria Limberis; Marcus Davey; Alan W Flake
Journal:  Mol Ther Methods Clin Dev       Date:  2014-09-10       Impact factor: 6.698

8.  Gene Therapy: The View from NCATS.

Authors:  Philip J Brooks; N Nora Yang; Christopher P Austin
Journal:  Hum Gene Ther       Date:  2016-01       Impact factor: 5.695

Review 9.  The identification of hematopoietic-specific regulatory elements for WASp gene expression.

Authors:  Jun Zhan; Irudayam Maria Johnson; Matthew Wielgosz; Arthur W Nienhuis
Journal:  Mol Ther Methods Clin Dev       Date:  2016-12-14       Impact factor: 6.698

Review 10.  Gene therapy of inherited retinal degenerations: prospects and challenges.

Authors:  Ivana Trapani; Sandro Banfi; Francesca Simonelli; Enrico M Surace; Alberto Auricchio
Journal:  Hum Gene Ther       Date:  2015-04       Impact factor: 5.695
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