Search research articles

ABOUT JoVE

Overview Leadership Blog JoVE Help Center

AUTHORS

Publishing Process Editorial Board Scope & Policies Peer Review FAQ Submit

LIBRARIANS

Testimonials Subscriptions Access Resources Library Advisory Board FAQ

RESEARCH

JoVE Journal Methods Collections JoVE Encyclopedia of Experiments Archive

EDUCATION

JoVE Core JoVE Business JoVE Science Education JoVE Lab Manual Faculty Resource Center Faculty Site

Terms & Conditions of Use

Search research articles

Related Experiment Videos

Parvovirus-based vectors for human gene therapy

¹Department of Medicine, Indiana University of School of Medicine, Indianapolis 46202-5120, USA.

Blood Cells

|January 1, 1994

Summary

Adeno-associated virus 2 (AAV) vectors offer a promising, nonpathogenic alternative for gene therapy. These vectors enable efficient transduction of hematopoietic stem cells without pre-stimulation, potentially improving transplantation outcomes.

Related Concept Videos

You might also read

Related Articles

Articles linked to this work by shared authors, journal, and citation graph.

Sort by

Same author

Dengue virus infection in renal allograft recipients: a case series during 2010 outbreak.

Transplant infectious disease : an official journal of the Transplantation Society·2012

Same author

Metal-insulator transition in SrTiO(3-x) thin films induced by frozen-out carriers.

Physical review letters·2011

Same author

Disruption of a mitochondrial protease machinery in Plasmodium falciparum is an intrinsic signal for parasite cell death.

Cell death & disease·2011

Same author

Social interaction and neuroses (the family and the primary group): a pilot study.

Indian journal of psychiatry·2011

Same author

Indigenous therapy in practice of psychiatry in India.

Indian journal of psychiatry·2011

Same author

The relation between depression and parkin genotype: the CORE-PD study.

Parkinsonism & related disorders·2011

Area of Science:

Gene Therapy
Virology
Hematopoietic Stem Cell Biology

Background:

Retroviral vectors are commonly used in gene therapy but have limitations.
Adeno-associated virus 2 (AAV), a human parvovirus, is nonpathogenic and integrates specifically into the human genome.
Current methods for hematopoietic stem cell transduction often require cytokine pre-stimulation, which can induce differentiation.

Purpose of the Study:

To evaluate adeno-associated virus 2 (AAV) vectors as an alternative to retroviral vectors in human gene therapy.
To assess the efficiency of recombinant AAV vectors for transducing slow- or non-cycling primary hematopoietic stem and progenitor cells.
To determine if AAV vectors can achieve high-efficiency transduction without cytokine pre-stimulation.

Main Methods:

Utilized a recombinant adeno-associated virus 2 (AAV) vector system.
Transduced slow- or non-cycling primary hematopoietic stem and progenitor cells.
Assessed transduction efficiency without prior cytokine stimulation.

Main Results:

Recombinant AAV vectors achieved high-efficiency transduction of target cells.
Transduction was successful in slow- or non-cycling hematopoietic stem and progenitor cells.
Cytokine pre-stimulation was not required for efficient transduction.

Conclusions:

Adeno-associated virus 2 (AAV) vectors represent a viable and potentially superior alternative to retroviral vectors for gene therapy applications.
The nonpathogenic nature and site-specific integration of AAV contribute to its therapeutic potential.
AAV vector systems facilitate efficient gene transfer into hematopoietic stem cells, circumventing pre-stimulation-induced differentiation and improving transplantation prospects.

Related Experiment Videos