Complementation of helper-dependent adenoviral vectors: size

Alemany, Ramón; Dai, Yifan; Lou, Yan; Sethi, Erica; Prokopenko, Elena; Josephs, Steven F.; Zhang, Weiwei

doi:10.1016/s0166-0934(97)00129-8

Cited by 38 publications

(43 citation statements)

References 24 publications

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“…219 However, a similar study on a series of mini-Ads with a progressive deletion of the Ad5 genome demonstrated a continual decrease of packaging efficiency from mini-Ad vectors with a genome size ranging from 32 kb to 6.5 kb. 216 These studies indicate that an optimal genome size (Ͼ75% of wt Ad genome) should be used for mini-Ad vectors to obtain high titers of the vectors.…”

Section: General Design Of Mini-ad Vectorsmentioning

confidence: 92%

“…Selective disabling of the packaging of the helper Ad became a logical solution. The technical approaches used to complete this strategy have primarily been reported in two aspects, a packaging signal partial deletion [215][216][217] and conditional deletion, 47,218 although other designs to manipulate the packaging signal or packaging process of the helper virus can also be considered, such as a synthetic packaging signal, packaging blockage by specific DNA-binding, packaging signal relocation, etc.…”

Section: Packaging-attenuated Ad As a Helpermentioning

confidence: 99%

“…So far, two groups have reported on the generation and characterization of the helper Ad with packaging signal mutations based on Hearing's constructs. [215][216][217] One helper (SV5) based on the dl309-267/358, 34 has a 91-bp deletion within the packaging signal and an E1A deletion (480 -1339 bp) with the rest of wt Ad5 genome. 215 The other helper Ad (AdH␤), based on dl309-194/243:274/358, 35 has packaging signal partial deletions (194 -243 and 274 -358 bp) and a complete E1 deletion (452-3328 bp).…”

Section: Packaging-attenuated Ad As a Helpermentioning

confidence: 99%

“…215 The other helper Ad (AdH␤), based on dl309-194/243:274/358, 35 has packaging signal partial deletions (194 -243 and 274 -358 bp) and a complete E1 deletion (452-3328 bp). 216,217 Both helper Ads can be propagated as single viruses in E1-complementing helper cells at a lower (ϳ100-fold less) efficiency than the E1-deleted-type viruses and have successfully supported the generation and propagation of mini-Ad vectors.…”

Section: Packaging-attenuated Ad As a Helpermentioning

confidence: 99%

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Development and application of adenoviral vectors for gene therapy of cancer

Zhang¹

1999

Cancer Gene Ther

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165

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For successful gene vaccination and therapy of cancer, it is essential to develop gene delivery vectors that can meet clinical and social requirements. The need for improved vectors was clearly manifested during the peak of the first wave of gene therapy. Adenoviral (Ad) vectors have recently drawn the attention of many of those involved in the field of gene therapy for cancer because of their practical advantages and application potential. Many experiments, innovations, preclinical studies, and clinical trials have generated an overwhelming amount of data and literature concerning this vector system. It is hoped that the comprehensive review presented here, which includes the principles, potential, capacity, and limitations of the current Ad systems, will help to further the rational development of the system. The literature in this article is organized in an attempt to emphasize Ad vector development in the aspects of technical approaches, practical hurdles and strategies to overcome them, significant experimental results, recent advances, future directions, etc. The technical range of this review covers details that are intended to serve as a reference for advanced technical readers and provide a foundation for initiates in the field of Ad vector gene therapy. The material presented here is also intended for nontechnical persons who want to have an overview of the significance and potential of the technology.

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Section: General Design Of Mini-ad Vectorsmentioning

confidence: 92%

Section: Packaging-attenuated Ad As a Helpermentioning

confidence: 99%

Section: Packaging-attenuated Ad As a Helpermentioning

confidence: 99%

Section: Packaging-attenuated Ad As a Helpermentioning

confidence: 99%

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Development and application of adenoviral vectors for gene therapy of cancer

Zhang¹

1999

Cancer Gene Ther

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165

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“…[10][11][12][13][14] As therapeutic expression cassettes usually do not reach up to 36 kb, there is a need to use stuffer DNA in order to complete the genome size for encapsidation. Initially, it was believed that stuffer DNA's unique role was the participation in the packaging of the vector genome.…”

Section: Introductionmentioning

confidence: 99%

Gutless adenovirus: last-generation adenovirus for gene therapy

2005

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Last-generation adenovirus vectors, also called helper-dependent or gutless adenovirus, are very attractive for gene therapy because the associated in vivo immune response is highly reduced compared to first-and second-generation adenovirus vectors, while maintaining high transduction efficiency and tropism. Nowadays, gutless adenovirus is administered in different organs, such as the liver, muscle or the central nervous system achieving high-level and long-term transgene expression in rodents and primates. However, as devoid of all viral coding regions, gutless vectors require viral proteins supplied in trans by a helper virus. To remove contamination by a helper virus from the final preparation, different systems based on the excision of the helper-packaging signal have been generated. Among them, Cre-loxP system is mostly used, although contamination levels still are 0.1-1% too high to be used in clinical trials. Recently developed strategies to avoid/reduce helper contamination were reviewed. Gene Therapy (2005) 12, S18-S27.

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Prospects for the treatment of phenylketonuria by gene therapy

Eisensmith

Kuzmin

Krougliak

1999

Ment. Retard. Dev. Disabil. Res. Rev.

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Gene therapy is being explored as a means of treating a variety of genetic diseases. Because of the central role of the liver in many metabolic pathways, and the relative ease with which foreign genes can be delivered to hepatocytes, monogenic disorders that result in deficiencies of liver‐specific proteins will be among the first targets of somatic gene therapy. Phenylketonuria represents an ideal model system in which to validate many of the principles of somatic gene therapy. This article will review previous efforts in the development of gene therapy for phenylketonuria using recombinant viral vectors, and provide an update of the recent progress that has been made in the development of the viral vector systems that may prove useful in these efforts. MRDD Research Reviews 1999;5:136–143. © 1999 Wiley‐Liss, Inc.

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Complementation of helper-dependent adenoviral vectors: size

Cited by 38 publications

References 24 publications

Development and application of adenoviral vectors for gene therapy of cancer

Development and application of adenoviral vectors for gene therapy of cancer

Gutless adenovirus: last-generation adenovirus for gene therapy

Prospects for the treatment of phenylketonuria by gene therapy

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