Literature DB >> 24773122

Gene therapy: charting a future course--summary of a National Institutes of Health Workshop, April 12, 2013.

Marina O'Reilly1, Howard J Federoff, Yuman Fong, Donald B Kohn, Amy P Patterson, Nabil Ahmed, Aravind Asokan, Shannon E Boye, Ronald G Crystal, Satiro De Oliveira, Linda Gargiulo, Scott Q Harper, Yasuhiro Ikeda, Robert Jambou, Maureen Montgomery, Lawrence Prograis, Eugene Rosenthal, Daniel H Sterman, Luk H Vandenberghe, Laurie Zoloth, Mehrdad Abedi, Jennifer Adair, Prasad S Adusumilli, William F Goins, Jhanelle Gray, Paul Monahan, Leslie Popplewell, Miguel Sena-Esteves, Bakhos Tannous, Thomas Weber, William Wierda, Rashmi Gopal-Srivastava, Cheryl L McDonald, Daniel Rosenblum, Jacqueline Corrigan-Curay.   

Abstract

Recently, the gene therapy field has begun to experience clinical successes in a number of different diseases using various approaches and vectors. The workshop Gene Therapy: Charting a Future Course, sponsored by the National Institutes of Health (NIH) Office of Biotechnology Activities, brought together early and mid-career researchers to discuss the key scientific challenges and opportunities, ethical and communication issues, and NIH and foundation resources available to facilitate further clinical advances.

Mesh：

Year:  2014        PMID： 24773122      PMCID： PMC4064731          DOI： 10.1089/hum.2014.045

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


  46 in total

Review 1.  Artificial T-cell receptors.

Authors:  M Pule; H Finney; A Lawson
Journal:  Cytotherapy       Date:  2003       Impact factor: 5.414

2.  An unparalleled engine for discovery and clinical introduction: the clinical and translational science awards and gene therapy.

Authors:  Pamela B Davis
Journal:  Hum Gene Ther       Date:  2012-03       Impact factor: 5.695

3.  Long-term preservation of cone photoreceptors and restoration of cone function by gene therapy in the guanylate cyclase-1 knockout (GC1KO) mouse.

Authors:  Sanford L Boye; Thomas Conlon; Kirsten Erger; Renee Ryals; Andy Neeley; Travis Cossette; Jijing Pang; Frank M Dyka; William W Hauswirth; Shannon E Boye
Journal:  Invest Ophthalmol Vis Sci       Date:  2011-09-09       Impact factor: 4.799

4.  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

5.  Allelic exclusion and peripheral reconstitution by TCR transgenic T cells arising from transduced human hematopoietic stem/progenitor cells.

Authors:  Francesca Giannoni; Cinnamon L Hardee; Jennifer Wherley; Eric Gschweng; Shantha Senadheera; Michael L Kaufman; Rebecca Chan; Ingrid Bahner; Vivian Gersuk; Xiaoyan Wang; David Gjertson; David Baltimore; Owen N Witte; James S Economou; Antoni Ribas; Donald B Kohn
Journal:  Mol Ther       Date:  2013-02-05       Impact factor: 11.454

6.  Efficacy and toxicity management of 19-28z CAR T cell therapy in B cell acute lymphoblastic leukemia.

Authors:  Marco L Davila; Isabelle Riviere; Xiuyan Wang; Shirley Bartido; Jae Park; Kevin Curran; Stephen S Chung; Jolanta Stefanski; Oriana Borquez-Ojeda; Malgorzata Olszewska; Jinrong Qu; Teresa Wasielewska; Qing He; Mitsu Fink; Himaly Shinglot; Maher Youssif; Mark Satter; Yongzeng Wang; James Hosey; Hilda Quintanilla; Elizabeth Halton; Yvette Bernal; Diana C G Bouhassira; Maria E Arcila; Mithat Gonen; Gail J Roboz; Peter Maslak; Dan Douer; Mark G Frattini; Sergio Giralt; Michel Sadelain; Renier Brentjens
Journal:  Sci Transl Med       Date:  2014-02-19       Impact factor: 17.956

7.  Long-term expression and safety of administration of AAVrh.10hCLN2 to the brain of rats and nonhuman primates for the treatment of late infantile neuronal ceroid lipofuscinosis.

Authors:  Dolan Sondhi; Linda Johnson; Keith Purpura; Sebastien Monette; Mark M Souweidane; Michael G Kaplitt; Barry Kosofsky; Kaleb Yohay; Douglas Ballon; Jonathan Dyke; Stephen M Kaminksy; Neil R Hackett; Ronald G Crystal
Journal:  Hum Gene Ther Methods       Date:  2012-11-06       Impact factor: 2.396

8.  Adenovirus-associated virus vector-mediated gene transfer in hemophilia B.

Authors:  Amit C Nathwani; Edward G D Tuddenham; Savita Rangarajan; Cecilia Rosales; Jenny McIntosh; David C Linch; Pratima Chowdary; Anne Riddell; Arnulfo Jaquilmac Pie; Chris Harrington; James O'Beirne; Keith Smith; John Pasi; Bertil Glader; Pradip Rustagi; Catherine Y C Ng; Mark A Kay; Junfang Zhou; Yunyu Spence; Christopher L Morton; James Allay; John Coleman; Susan Sleep; John M Cunningham; Deokumar Srivastava; Etiena Basner-Tschakarjan; Federico Mingozzi; Katherine A High; John T Gray; Ulrike M Reiss; Arthur W Nienhuis; Andrew M Davidoff
Journal:  N Engl J Med       Date:  2011-12-10       Impact factor: 176.079

9.  RNAi-mediated Gene Silencing of Mutant Myotilin Improves Myopathy in LGMD1A Mice.

Authors:  Jian Liu; Lindsay M Wallace; Sara E Garwick-Coppens; Darcée D Sloboda; Carol S Davis; Chady H Hakim; Michael A Hauser; Susan V Brooks; Jerry R Mendell; Scott Q Harper
Journal:  Mol Ther Nucleic Acids       Date:  2014-04-29       Impact factor: 10.183

10.  Combinational targeting offsets antigen escape and enhances effector functions of adoptively transferred T cells in glioblastoma.

Authors:  Meenakshi Hegde; Amanda Corder; Kevin K H Chow; Malini Mukherjee; Aidin Ashoori; Yvonne Kew; Yi Jonathan Zhang; David S Baskin; Fatima A Merchant; Vita S Brawley; Tiara T Byrd; Simone Krebs; Meng Fen Wu; Hao Liu; Helen E Heslop; Stephen Gottschalk; Stephen Gottachalk; Eric Yvon; Nabil Ahmed
Journal:  Mol Ther       Date:  2013-08-13       Impact factor: 11.454
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  5 in total

Review 1.  Let There Be Light: Gene and Cell Therapy for Blindness.

Authors:  Deniz Dalkara; Olivier Goureau; Katia Marazova; José-Alain Sahel
Journal:  Hum Gene Ther       Date:  2016-02       Impact factor: 5.695

2.  Long-term rescue of cone photoreceptor degeneration in retinitis pigmentosa 2 (RP2)-knockout mice by gene replacement therapy.

Authors:  Suddhasil Mookherjee; Suja Hiriyanna; Kayleigh Kaneshiro; Linjing Li; Yichao Li; Wei Li; Haohua Qian; Tiansen Li; Hemant Khanna; Peter Colosi; Anand Swaroop; Zhijian Wu
Journal:  Hum Mol Genet       Date:  2015-09-10       Impact factor: 6.150

3.  Stability and toxicity of empty or gene-loaded lipopolysaccharide-amine nanopolymersomes.

Authors:  Qinmei Wang; Ying Chen; Lichun Wang; Xinchun Zhang; Hongzhang Huang; Wei Teng
Journal:  Int J Nanomedicine       Date:  2015-01-13

4.  Employing a gain-of-function factor IX variant R338L to advance the efficacy and safety of hemophilia B human gene therapy: preclinical evaluation supporting an ongoing adeno-associated virus clinical trial.

Authors:  Paul E Monahan; Junjiang Sun; Tong Gui; Genlin Hu; William B Hannah; David G Wichlan; Zhijian Wu; Joshua C Grieger; Chengwen Li; Thipparat Suwanmanee; Darrel W Stafford; Carmen J Booth; Jade J Samulski; Tal Kafri; Scott W J McPhee; R Jude Samulski
Journal:  Hum Gene Ther       Date:  2015-01-21       Impact factor: 5.695

5.  Mesoangioblast delivery of miniagrin ameliorates murine model of merosin-deficient congenital muscular dystrophy type 1A.

Authors:  Teuta Domi; Emanuela Porrello; Daniele Velardo; Alessia Capotondo; Alessandra Biffi; Rossana Tonlorenzi; Stefano Amadio; Alessandro Ambrosi; Yuko Miyagoe-Suzuki; Shin'ichi Takeda; Markus A Ruegg; Stefano Carlo Previtali
Journal:  Skelet Muscle       Date:  2015-09-03       Impact factor: 4.912
  5 in total

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