Literature DB >> 18288209

Progress and prospects: gene therapy for genetic diseases with helper-dependent adenoviral vectors.

N Brunetti-Pierri1, P Ng.   

Abstract

Preclinical studies in small and large animal models using helper-dependent adenoviral vectors (HDAds) have generated promising results for the treatment of genetic diseases. However, clinical translation is complicated by the dose-dependent, capsid-mediated acute toxic response following systemic vector injection. With the advancements in vectorology, a better understanding of vector-mediated toxicity, and improved delivery methods, HDAds may emerge as an important vector for gene therapy of genetic diseases and this report highlights recent progress and prospects in this field.

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Year:  2008        PMID： 18288209      PMCID： PMC3668677          DOI： 10.1038/gt.2008.14

Source DB:  PubMed          Journal:  Gene Ther        ISSN： 0969-7128            Impact factor:   5.250


  49 in total

Review 1.  Helper-dependent adenoviral vectors for gene therapy.

Authors:  Donna J Palmer; Philip Ng
Journal:  Hum Gene Ther       Date:  2005-01       Impact factor: 5.695

2.  Increased hepatic transduction with reduced systemic dissemination and proinflammatory cytokines following hydrodynamic injection of helper-dependent adenoviral vectors.

Authors:  Nicola Brunetti-Pierri; Donna J Palmer; Viraj Mane; Milton Finegold; Arthur L Beaudet; Philip Ng
Journal:  Mol Ther       Date:  2005-07       Impact factor: 11.454

3.  Aerosol delivery of an enhanced helper-dependent adenovirus formulation to rabbit lung using an intratracheal catheter.

Authors:  David R Koehler; Helena Frndova; Kitty Leung; Emily Louca; Donna Palmer; Philip Ng; Colin McKerlie; Peter Cox; Allan L Coates; Jim Hu
Journal:  J Gene Med       Date:  2005-11       Impact factor: 4.565

4.  Characterization of hemodynamic events following intravascular infusion of recombinant adenovirus reveals possible solutions for mitigating cardiovascular responses.

Authors:  Todd Machemer; Heidrun Engler; Van Tsai; Seoju Lee; Susan Cannon-Carlson; Marcio Voloch; Thomas Schluep; Sundari Ravindran; Gary Vellekamp; Elena Brin; Douglas Cornell; Suganto Sutjipto; Shu Fen Wen; Mark Horn; Nico Van Rooijen; Dan Maneval; Beth Hutchins; Drake LaFace
Journal:  Mol Ther       Date:  2005-08       Impact factor: 11.454

5.  Sustained phenotypic correction of canine hemophilia B after systemic administration of helper-dependent adenoviral vector.

Authors:  Nicola Brunetti-Pierri; Timothy C Nichols; Stephanie McCorquodale; Elizabeth Merricks; Donna J Palmer; Arthur L Beaudet; Philip Ng
Journal:  Hum Gene Ther       Date:  2005-07       Impact factor: 5.695

6.  Local delivery of a viral vector mitigates neutralization by antiviral antibodies and results in efficient transduction of rabbit liver.

Authors:  Bradley L Hodges; Kristin M Taylor; Qiuming Chu; Samantha E Scull; Rebecca G Serriello; Scott C Anderson; Fei Wang; Ronald K Scheule
Journal:  Mol Ther       Date:  2005-08-31       Impact factor: 11.454

7.  Evaluation of polyethylene glycol modification of first-generation and helper-dependent adenoviral vectors to reduce innate immune responses.

Authors:  Hoyin Mok; Donna J Palmer; Philip Ng; Michael A Barry
Journal:  Mol Ther       Date:  2005-01       Impact factor: 11.454

8.  PEGylated helper-dependent adenoviral vectors: highly efficient vectors with an enhanced safety profile.

Authors:  M A Croyle; H T Le; K D Linse; V Cerullo; G Toietta; A Beaudet; L Pastore
Journal:  Gene Ther       Date:  2005-04       Impact factor: 5.250

9.  Lifelong elimination of hyperbilirubinemia in the Gunn rat with a single injection of helper-dependent adenoviral vector.

Authors:  Gabriele Toietta; Viraj P Mane; Wilma S Norona; Milton J Finegold; Philip Ng; Antony F McDonagh; Arthur L Beaudet; Brendan Lee
Journal:  Proc Natl Acad Sci U S A       Date:  2005-03-07       Impact factor: 11.205

10.  The size of sinusoidal fenestrae is a critical determinant of hepatocyte transduction after adenoviral gene transfer.

Authors:  J Lievens; J Snoeys; K Vekemans; S Van Linthout; R de Zanger; D Collen; E Wisse; B De Geest
Journal:  Gene Ther       Date:  2004-10       Impact factor: 5.250
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  24 in total

1.  DRG-targeted helper-dependent adenoviruses mediate selective gene delivery for therapeutic rescue of sensory neuronopathies in mice.

Authors:  Tomoya Terashima; Kazuhiro Oka; Angelika B Kritz; Hideto Kojima; Andrew H Baker; Lawrence Chan
Journal:  J Clin Invest       Date:  2009-07       Impact factor: 14.808

2.  Limitations of Adenoviral Vector-Mediated Delivery of Gold Nanoparticles to Tumors for Hyperthermia Induction.

Authors:  Vaibhav Saini; Dmitri V Martyshki; Victoria D Towner; Sergey B Mirov; Maaike Everts
Journal:  Open Nanomed J       Date:  2009

3.  A rapid Q-PCR titration protocol for adenovirus and helper-dependent adenovirus vectors that produces biologically relevant results.

Authors:  Sean D Gallaher; Arnold J Berk
Journal:  J Virol Methods       Date:  2013-04-26       Impact factor: 2.014

4.  Helper-dependent adenovirus achieve more efficient and persistent liver transgene expression in non-human primates under immunosuppression.

Authors:  C Unzu; I Melero; S Hervás-Stubbs; A Sampedro; U Mancheño; A Morales-Kastresana; I Serrano-Mendioroz; R E de Salamanca; A Benito; A Fontanellas
Journal:  Gene Ther       Date:  2015-07-23       Impact factor: 5.250

Review 5.  Antioxidant enzyme gene transfer for ischemic diseases.

Authors:  Jian Wu; James G Hecker; Nipavan Chiamvimonvat
Journal:  Adv Drug Deliv Rev       Date:  2009-02-20       Impact factor: 15.470

Review 6.  Prospects, promise and problems on the road to effective vaccines and related therapies for substance abuse.

Authors:  Stephen Brimijoin; Xiaoyun Shen; Frank Orson; Thomas Kosten
Journal:  Expert Rev Vaccines       Date:  2013-03       Impact factor: 5.217

Review 7.  Barriers to inhaled gene therapy of obstructive lung diseases: A review.

Authors:  Namho Kim; Gregg A Duncan; Justin Hanes; Jung Soo Suk
Journal:  J Control Release       Date:  2016-05-16       Impact factor: 9.776

8.  Enhanced anti-tumor effects of combined MDR1 RNA interference and human sodium/iodide symporter (NIS) radioiodine gene therapy using an adenoviral system in a colon cancer model.

Authors:  S J Ahn; Y H Jeon; Y J Lee; Y L Lee; S-W Lee; B-C Ahn; J-H Ha; J Lee
Journal:  Cancer Gene Ther       Date:  2010-02-26       Impact factor: 5.987

9.  Capsid-modified adenoviral vectors for improved muscle-directed gene therapy.

Authors:  Kilian Guse; Masataka Suzuki; Gautam Sule; Terry K Bertin; Henna Tyynismaa; Sofia Ahola-Erkkilä; Donna Palmer; Anu Suomalainen; Philip Ng; Vincenzo Cerullo; Akseli Hemminki; Brendan Lee
Journal:  Hum Gene Ther       Date:  2012-08-13       Impact factor: 5.695

10.  Gene therapy for inborn errors of liver metabolism: progress towards clinical applications.

Authors:  Nicola Brunetti-Pierri
Journal:  Ital J Pediatr       Date:  2008-11-18       Impact factor: 2.638
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