Correction of Cardiac Abnormalities in Fabry Mice by Direct Intraventricular Injection of a Recombinant Lentiviral Vector That Engineers Expression of .ALPHA.-Galactosidase A

Yoshimitsu, Makoto; Higuchi, Koji; Dawood, Fayez; Rasaiah, Vanessa I.; Ayach, Bilal; Chen, Manyin; Liu, Peter; Medin, Jeffrey A.

doi:10.1253/circj.70.1503

Cited by 29 publications

(24 citation statements)

References 30 publications

(21 reference statements)

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“…Although these vectors are capable of transducing 80 -100% of cardiac myocytes in vitro (71,588,779,780), in vivo efficiencies rarely achieve a transduction efficiency of above 30% (71,238). For cardiac expression, direct injection is by far the most efficient means of delivery, with little expression seen after vascular delivery (996). However, in cardiac transplant rejection studies, this relatively low level of expression has yielded significant results (1008).…”

Section: Retroviral Vectorsmentioning

confidence: 99%

Designing Heart Performance by Gene Transfer

Davis¹,

Westfall²,

Townsend³

et al. 2008

Physiological Reviews

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The birth of molecular cardiology can be traced to the development and implementation of high-fidelity genetic approaches for manipulating the heart. Recombinant viral vector-based technology offers a highly effective approach to genetically engineer cardiac muscle in vitro and in vivo. This review highlights discoveries made in cardiac muscle physiology through the use of targeted viral-mediated genetic modification. Here the history of cardiac gene transfer technology and the strengths and limitations of viral and nonviral vectors for gene delivery are reviewed. A comprehensive account is given of the application of gene transfer technology for studying key cardiac muscle targets including Ca2+handling, the sarcomere, the cytoskeleton, and signaling molecules and their posttranslational modifications. The primary objective of this review is to provide a thorough analysis of gene transfer studies for understanding cardiac physiology in health and disease. By comparing results obtained from gene transfer with those obtained from transgenesis and biophysical and biochemical methodologies, this review provides a global view of cardiac structure-function with an eye towards future areas of research. The data presented here serve as a basis for discovery of new therapeutic targets for remediation of acquired and inherited cardiac diseases.

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Section: Retroviral Vectorsmentioning

confidence: 99%

Designing Heart Performance by Gene Transfer

Davis¹,

Westfall²,

Townsend³

et al. 2008

Physiological Reviews

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show abstract

“…Moreover, it has to be mentioned that for immunotherapy, the lentiviral vectors are systemically administered, whereas in the case of gene therapy, the lentiviral vectors are most often administered in the Lentiviral vectors in cancer immunotherapy K Breckpot et al tissue of interest, resulting in transduction of these tissue-specific cells. [157][158][159][160][161] Second, when using ex vivo-transduced DC or targeting DC in vivo, terminally differentiated, nondividing cells are transduced as opposed to extensively proliferating cells in the SCID trial, thereby posing less of a risk for oncogenic transformations. Finally, when an immune response is induced in vivo, the effector cells will most likely kill cells that express the target antigen, including the transduced APC, thus, further reducing the risk for oncogenesis.…”

Section: Concerns In View Of Lentiviral Vectors As Anticancer Vaccinementioning

confidence: 99%

Lentiviral vectors for cancer immunotherapy: transforming infectious particles into therapeutics

2007

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Lentiviral vectors have emerged as promising tools for both gene therapy and immunotherapy purposes. They exhibit several advantages over other viral systems in that they are less immunogenic and are capable of transducing a wide range of different cell types, including dendritic cells (DC). DC transduced ex vivo with a whole range of different (tumor) antigens were capable of inducing strong antigen-specific T-cell responses, both in vitro and in vivo. Recently, the administration of lentiviral vectors in vivo has gained substantial interest as an alternative method for antigenspecific immunization. This method offers a number of advantages over DC vaccines as the same lentivirus can in principle be used for all patients resulting in a significantly reduced cost and requirement for considerably less expertise for the generation and administration of lentiviral vaccines. By selectively targeting lentiviral vectors to, or restricting transgene expression in certain cell types, selectivity, safety and efficacy can be further improved. This review will focus on the use of direct administration of lentiviral vectors encoding tumor-associated antigens (TAA) for the induction of tumor-specific immune responses in vivo, with a special focus on problems related to the generation of large amounts of highly purified virus and specific targeting of antigenpresenting cells (APC).

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“…Hybrid "pseudotyped" lentivirus have been produced to expand their tropism for other cell types. In the context of transfecting cardiomyocytes, lentiviral-based vectors are as effecient as adenoviruses, with transgene expression lasting longer (Yoshimitsu 2006). They can incorporate constructs up to 8 kB in size (Yoshimitsu).…”

Section: Viral Vectorsmentioning

confidence: 99%

Gene Therapy of the Heart through Targeting Non-Cardiac Cells

Valen¹

2011

Targets in Gene Therapy

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Correction of Cardiac Abnormalities in Fabry Mice by Direct Intraventricular Injection of a Recombinant Lentiviral Vector That Engineers Expression of .ALPHA.-Galactosidase A

Cited by 29 publications

References 30 publications

Designing Heart Performance by Gene Transfer

Designing Heart Performance by Gene Transfer

Lentiviral vectors for cancer immunotherapy: transforming infectious particles into therapeutics

Gene Therapy of the Heart through Targeting Non-Cardiac Cells

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