Viral Vectors for Gene Therapy

Robbins, Paul D.; Ghivizzani, Steven C.

doi:10.1016/s0163-7258(98)00020-5

Cited by 466 publications

(228 citation statements)

References 60 publications

Supporting

Mentioning

211

Contrasting

Unclassified

Order By: Relevance

“…5, n = 6). As reported by others, transduction of muscle fibers by AAV vectors appeared later and lasted longer than that obtained with ADV vectors (Robbins and Ghivizzani 1998). In mouse soleus, we found strong EGFP transduction 6 months after the injection, which was the last time point we examined.…”

Section: Transduction Of Muscle Fibers By Aav Vectorssupporting

confidence: 81%

“…The smaller size of the AAV vector should favor rather than disfavor uptake. Presumably, muscle fibers and motoneurons of different types express different receptors to which ADV or AAV vectors must bind before they can enter the cell (Robbins and Ghivizzani 1998;Summerford and Samulski 1998;Qing et al 1999;Summerford et al 1999;Pruchnic et al 2000).…”

Section: Fig 5a-fmentioning

confidence: 99%

“…These viral vectors are known to transduce post-mitotic cells such as neurons and muscle fibers, and offer higher efficiencies than conventional non-viral transfection systems (Robbins and Ghivizzani 1998;Björklund et al 2000). However, a number of uncertainties remain concerning their efficiencies in different cell populations.…”

Section: Introductionmentioning

confidence: 99%

See 2 more Smart Citations

Targeting functional subtypes of spinal motoneurons and skeletal muscle fibers in vivo by intramuscular injection of adenoviral and adeno-associated viral vectors

Martinov

Sefland

Walaas

et al. 2002

Anatomy and Embryology

View full text Add to dashboard Cite

We report that functional subtypes of spinal motoneurons and skeletal muscle fibers can be selectively transduced using replication-defective adenoviral (ADV) or adeno-associated (AAV) viral vectors. After intramuscular injection in adult rodents, ADV vectors transduced both fast-twitch and slow-twitch skeletal muscle fibers. Intramuscular injection of ADV vectors also caused transduction of spinal motoneurons and dorsal root ganglion cells. However, only neurons innervating the injected muscle were transduced, as shown by co-injection of a retrograde axonal tracer. In adult male rats it is therefore possible to transduce fast or slow spinal motoneurons and muscle fibers selectively since in these animals, the extensor digitorum longus and soleus muscles contain almost exclusively fast or slow motor units, respectively. In rats, AAV vectors transduced muscle fibers in the predominantly fast extensor digitorum longus but not in the predominantly slow soleus muscle. We did not observe any transduction of spinal motoneurons following intramuscular injection of AAV vectors. These results show that physiologically and clinically important subpopulations of cells in the neuromuscular system can be selectively transduced by viral vectors.

show abstract

Section: Transduction Of Muscle Fibers By Aav Vectorssupporting

confidence: 81%

Section: Fig 5a-fmentioning

confidence: 99%

See 1 more Smart Citation

Targeting functional subtypes of spinal motoneurons and skeletal muscle fibers in vivo by intramuscular injection of adenoviral and adeno-associated viral vectors

Martinov

Sefland

Walaas

et al. 2002

Anatomy and Embryology

View full text Add to dashboard Cite

show abstract

“…However, use of chemicals or liposomes as the gene delivery techniques, although convenient in in vitro experiments, may be restricted in clinical applications because of relatively low transfection efficiencies and other limitations. Viral vectors, on the other hand, have advantages in that they can be used to transduce quiescent cells such as hepatocytes, myocytes, and neurons and that the gene can be integrated into the DNA of the host cell so that it will be replicated (in dividing cells) and expressed indefinitely (35)(36)(37)(38). We chose recombinant AAV vector because AAV has not been implicated as the causative agent for any diseases, does not elicit antibodies against itself, and supports long term transgene expression (17,18,24).…”

Section: Figmentioning

confidence: 99%

Use of the NADH-Quinone Oxidoreductase (NDI1) Gene ofSaccharomyces cerevisiae as a Possible Cure for Complex I Defects in Human Cells

Seo¹,

Wang²,

Flotte³

et al. 2000

Journal of Biological Chemistry

View full text Add to dashboard Cite

The Ndi1 enzyme of Saccharomyces cerevisiae is a single subunit rotenone-insensitive NADH-quinone oxidoreductase that is located on the matrix side of the inner mitochondrial membrane. We have shown previously that the NDI1 (rAAV-NDI1). With rAAV-NDI1 as the gene delivery method, we were able to achieve high transduction efficiencies (nearly 100%) even in 143B cells that are difficult to transfect by lipofection or calcium phosphate precipitation methods. The NDI1 gene was successfully introduced into non-proliferating human cells using rAAV-NDI1. The expressed Ndi1 protein was shown to be functionally active just as seen for proliferating cells. Furthermore, when cells were cultured under the conditions where energy has to be provided by respiration, the NDI1-transduced cells were able to grow even in the presence of added complex I inhibitor such as rotenone and 1-methyl-4-phenylpyridinium ion. In contrast, control cells that did not receive the NDI1 gene failed to survive as anticipated. The Ndi1 protein has a great potential as a molecular remedy for complex I defects, and it is highly likely that the same strategy can be extended to correction of other mitochondrial disorders.Mitochondrial proton-translocating NADH-quinone (Q) 1 oxidoreductase (complex I) of bovine heart is composed of at least 43 distinct subunits (1, 2). Of these subunits, seven are encoded by mtDNA. Those are designated

show abstract

“…In both viruses, CN706 and CV764, the adenoviral E3 region, which is not essential for viral replication, has been deleted [84,85]. However, recent studies demonstrate that the proteins encoded by the E3 region might play a role in assisting virus release and evading or slowing host immune responses to the virus [87,88]. It was therefore of interest to construct an adenovirus which retains the entire E3 region, but replicates in a prostate specific manner: CV787 was generated using the rat probasin prostate-specific promoter, driving the E1A expression, and the prostate-specific enhancers controlling the E1B gene [85].…”

Section: Other Viruses Used As Oncolytic Agentsmentioning

confidence: 99%

Replication-selective viruses for cancer therapy

Biederer¹,

Ries

Brandts

et al. 2001

J Mol Med

View full text Add to dashboard Cite

Advances in our understanding of the molecular basis of cancer and the availability of technology to genetically engineer viruses have led to the development of replication-competent viruses to treat cancer. In theory, replication-selective viruses offer several appealing properties as biological agents for cancer therapy: they kill tumor cells selectively, and their replication leads to amplification of their oncolytic potential. Most preclinical experiments in tissue culture and in animal models support this notion. Clinical data on the first generation of replication-selective viruses are now rapidly accruing. The therapeutic index, and ultimately the clinical outcome, will depend on a complex balance between host and viral factors. This review discusses strategies to kill cancer cells based on our understanding of their molecular defects and the progress being made using replication-competent viruses for tumor therapy. We focus our discussion on a replication-selective adenovirus called ONYX-015 that has recently demonstrated encouraging results in clinical trials

show abstract

Viral Vectors for Gene Therapy

Cited by 466 publications

References 60 publications

Targeting functional subtypes of spinal motoneurons and skeletal muscle fibers in vivo by intramuscular injection of adenoviral and adeno-associated viral vectors

Targeting functional subtypes of spinal motoneurons and skeletal muscle fibers in vivo by intramuscular injection of adenoviral and adeno-associated viral vectors

Use of the NADH-Quinone Oxidoreductase (NDI1) Gene ofSaccharomyces cerevisiae as a Possible Cure for Complex I Defects in Human Cells

Replication-selective viruses for cancer therapy

Contact Info

Product

Resources

About