Literature DB >> 22974119

Center for fetal monkey gene transfer for heart, lung, and blood diseases: an NHLBI resource for the gene therapy community.

Alice F Tarantal1, Sonia I Skarlatos.   

Abstract

The goals of the National Heart, Lung, and Blood Institute (NHLBI) Center for Fetal Monkey Gene Transfer for Heart, Lung, and Blood Diseases are to conduct gene transfer studies in monkeys to evaluate safety and efficiency; and to provide NHLBI-supported investigators with expertise, resources, and services to actively pursue gene transfer approaches in monkeys in their research programs. NHLBI-supported projects span investigators throughout the United States and have addressed novel approaches to gene delivery; "proof-of-principle"; assessed whether findings in small-animal models could be demonstrated in a primate species; or were conducted to enable new grant or IND submissions. The Center for Fetal Monkey Gene Transfer for Heart, Lung, and Blood Diseases successfully aids the gene therapy community in addressing regulatory barriers, and serves as an effective vehicle for advancing the field.

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Year:  2012        PMID: 22974119      PMCID: PMC3498881          DOI: 10.1089/hum.2012.178

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


  58 in total

Review 1.  Therapy for beta-globinopathies: a brief review and determinants for successful and safe correction.

Authors:  Ajay Perumbeti; Punam Malik
Journal:  Ann N Y Acad Sci       Date:  2010-08       Impact factor: 5.691

2.  Overexpression of transforming growth factor-beta1 in fetal monkey lung results in prenatal pulmonary fibrosis.

Authors:  A F Tarantal; H Chen; T T Shi; C-H Lu; A B Fang; S Buckley; M Kolb; J Gauldie; D Warburton; W Shi
Journal:  Eur Respir J       Date:  2010-03-29       Impact factor: 16.671

Review 3.  Large animal models of hematopoietic stem cell gene therapy.

Authors:  G D Trobridge; H-P Kiem
Journal:  Gene Ther       Date:  2010-04-29       Impact factor: 5.250

Review 4.  Update on gene therapy for adenosine deaminase-deficient severe combined immunodeficiency.

Authors:  Francesca Ferrua; Immacolata Brigida; Alessandro Aiuti
Journal:  Curr Opin Allergy Clin Immunol       Date:  2010-12

5.  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

6.  Efficient and stable MGMT-mediated selection of long-term repopulating stem cells in nonhuman primates.

Authors:  Brian C Beard; Grant D Trobridge; Christina Ironside; Jeannine S McCune; Jennifer E Adair; Hans-Peter Kiem
Journal:  J Clin Invest       Date:  2010-06-14       Impact factor: 14.808

7.  AAV's anatomy: roadmap for optimizing vectors for translational success.

Authors:  Angela M Mitchell; Sarah C Nicolson; Jayme K Warischalk; R Jude Samulski
Journal:  Curr Gene Ther       Date:  2010-10       Impact factor: 4.391

8.  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

9.  Long-term luciferase expression monitored by bioluminescence imaging after adeno-associated virus-mediated fetal gene delivery in rhesus monkeys (Macaca mulatta).

Authors:  Alice F Tarantal; C Chang I Lee
Journal:  Hum Gene Ther       Date:  2010-02       Impact factor: 5.695

Review 10.  Update on gene therapy for immunodeficiencies.

Authors:  Donald B Kohn
Journal:  Clin Immunol       Date:  2010-01-13       Impact factor: 3.969

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  11 in total

Review 1.  Dr. Sonia Skarlatos and the National Heart, Lung, and Blood institute translational research and resource programs.

Authors:  Cheryl L McDonald
Journal:  Hum Gene Ther       Date:  2013-11       Impact factor: 5.695

2.  Transfer of Therapeutic Genes into Fetal Rhesus Monkeys Using Recombinant Adeno-Associated Type I Viral Vectors.

Authors:  Thomas J Conlon; Cathryn S Mah; Christina A Pacak; Mary B Rucker Henninger; Kirsten E Erger; Marda L Jorgensen; C Chang I Lee; Alice F Tarantal; Barry J Byrne
Journal:  Hum Gene Ther Clin Dev       Date:  2016-12       Impact factor: 5.032

3.  Myeloid-lymphoid ontogeny in the rhesus monkey (Macaca mulatta).

Authors:  Cynthia A Batchelder; Nadire Duru; Charles I Lee; Chris A R Baker; Louise Swainson; Joseph M Mccune; Alice F Tarantal
Journal:  Anat Rec (Hoboken)       Date:  2014-05-28       Impact factor: 2.064

4.  Effects of vector backbone and pseudotype on lentiviral vector-mediated gene transfer: studies in infant ADA-deficient mice and rhesus monkeys.

Authors:  Denise Carbonaro Sarracino; Alice F Tarantal; C Chang I Lee; Michele Martinez; Xiangyang Jin; Xiaoyan Wang; Cinnamon L Hardee; Sabine Geiger; Christoph A Kahl; Donald B Kohn
Journal:  Mol Ther       Date:  2014-06-13       Impact factor: 11.454

5.  Systemic and Persistent Muscle Gene Expression in Rhesus Monkeys with a Liver De-Targeted Adeno-Associated Virus Vector.

Authors:  Alice F Tarantal; C Chang I Lee; Michele L Martinez; Aravind Asokan; R Jude Samulski
Journal:  Hum Gene Ther       Date:  2017-01-25       Impact factor: 5.695

6.  Safe and Sustained Expression of Human Iduronidase After Intrathecal Administration of Adeno-Associated Virus Serotype 9 in Infant Rhesus Monkeys.

Authors:  Juliette Hordeaux; Christian Hinderer; Elizabeth L Buza; Jean-Pierre Louboutin; Tahsin Jahan; Peter Bell; Jessica A Chichester; Alice F Tarantal; James M Wilson
Journal:  Hum Gene Ther       Date:  2019-06-10       Impact factor: 5.695

Review 7.  Nonhuman Primates in Translational Research.

Authors:  Alice F Tarantal; Stephen C Noctor; Dennis J Hartigan-O'Connor
Journal:  Annu Rev Anim Biosci       Date:  2022-02-15       Impact factor: 13.341

8.  Development of operational immunologic tolerance with neonatal gene transfer in nonhuman primates: preliminary studies.

Authors:  D S Tai; C Hu; C C I Lee; M Martinez; G Cantero; E H Kim; A F Tarantal; G S Lipshutz
Journal:  Gene Ther       Date:  2015-08-23       Impact factor: 5.250

Review 9.  Adeno-associated virus-mediated gene therapy for metabolic myopathy.

Authors:  Cathryn S Mah; Meghan S Soustek; A Gary Todd; Angela McCall; Barbara K Smith; Manuela Corti; Darin J Falk; Barry J Byrne
Journal:  Hum Gene Ther       Date:  2013-11       Impact factor: 5.695

10.  Microglia: An Intrinsic Component of the Proliferative Zones in the Fetal Rhesus Monkey (Macaca mulatta) Cerebral Cortex.

Authors:  Nicole Barger; Janet Keiter; Anna Kreutz; Anjana Krishnamurthy; Cody Weidenthaler; Verónica Martínez-Cerdeño; Alice F Tarantal; Stephen C Noctor
Journal:  Cereb Cortex       Date:  2019-07-05       Impact factor: 5.357

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