Navigation Links
Novel reference material to standardize gene therapy applications

New Rochelle, NY, October 6, 2010The introduction of a new, fully characterized viral vector for use as reference material to help standardize gene therapy protocols in research applications and human clinical trials is described in an article in Human Gene Therapy, a peer-reviewed journal published by Mary Ann Liebert, Inc. ( The article, which is published online ahead of print, is available free online at

The growing popularity in the gene therapy community of using recombinant adeno-associated virus (rAAV) vectors as vehicles to transfer a gene of interest into a host cell has led to increasing numbers of human clinical trials and animal studies with rAAV. In the past, the absence of standardizationsuch as for rAAV potency and dosinghave made it difficult for researchers and clinicians to compare their protocols and the results of gene therapy experiments. The availability of this rAAV reference standard will allow vector parameters to be expressed in common units, vector doses administered by different investigators to be normalized to an agreed-upon standard, and laboratories to calibrate their internal standards to a common reference material.

Martin Lock, from the Gene Therapy Program at the University of Pennsylvania (Philadelphia) and colleagues from several major medical and research institutions around the world, describe the development of reference standard in the article "Characterization of a Recombinant Adeno-Associated Virus Type 2 Reference Standard Material."

The American Type Culture Collection will make the rAAV2 reference standard material available to the scientific community. AAV serotype 2 was selected as the basis for the reference material because it is the best characterized AAV subtype. The material has been thoroughly analyzed and found to be free of microbial contamination. During development of the rAAV2 reference standard samples were distributed to 16 laboratories worldwide for extensive analysis and characterization.

"The development of a reference standard for an AAV vector is an important step towards the eventual commercial development of this important vector platform," says James M. Wilson, MD, PhD, Editor-in-Chief of Human Gene Therapy, and Head of the Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania School of Medicine, in Philadelphia.


Contact: Vicki Cohn
Mary Ann Liebert, Inc./Genetic Engineering News

Related biology news :

1. Scripps Research scientists develop novel test that identifies river blindness
2. Interfering with a double-edged sword: novel anti-inflammatory functions for interferons
3. Novartis and collaborators discover novel antimalarial drug candidate
4. Researchers discover novel mechanism protecting plants against freezing
5. Novel antisense therapies protect primates from lethal Ebola and Marburg viruses
6. Novel bee venom derivative forms a nanoparticle smart bomb to target cancer cells
7. UM researcher identifies novel treatment for pain in sickle cell disease
8. Novel microfluidic HIV test is quick and cheap
9. NIH-supported study finds novel pathway may open doors for new blood pressure treatments
10. Novel protein essential for successful pregnancy
11. UT Southwestern researchers use novel sperm stem-cell technique to produce genetically modified rats
Post Your Comments:
Related Image:
Novel reference material to standardize gene therapy applications
(Date:11/12/2015)... , Nov. 11, 2015   Growing need ... analytical tools has been paving the way for ... determination of discrete analytes in clinical, agricultural, environmental, ... being predominantly used in medical applications, however, their ... sectors due to continuous emphasis on improving product ...
(Date:11/10/2015)... 2015 About signature verification ... to identify and verify the identity of an ... the secure and accurate method of authentication and ... individual because each individual,s signature is highly unique. ... dynamic signature of an individual is compared and ...
(Date:11/4/2015)... 4, 2015 --> ... published by Transparency Market Research "Home Security Solutions Market - ... 2015 - 2022", the global home security solutions market is expected ... 2022. The market is estimated to expand at a ... to 2022. Rising security needs among customers at homes, ...
Breaking Biology News(10 mins):
(Date:11/25/2015)... ... November 25, 2015 , ... ... were featured on AngelList early in their initial angel funding process. Now, they ... for individuals looking to make early stage investments in the microbiome space. ...
(Date:11/24/2015)... Nov. 24, 2015 Halozyme Therapeutics, Inc. (NASDAQ: HALO ... New York on Wednesday, December 2 at ... , president and CEO, will provide a corporate overview. ... at 1:00 p.m. ET/10:00 a.m. PT . ... will provide a corporate overview. --> th Annual ...
(Date:11/24/2015)... /PRNewswire/ - Aeterna Zentaris Inc. (NASDAQ:  AEZS) (TSX: AEZ) (the ... the Toronto Stock Exchange, confirms that as of the ... developments that would cause the recent movements in the ... --> About Aeterna Zentaris Inc. ... Aeterna Zentaris is a specialty biopharmaceutical company engaged in ...
(Date:11/24/2015)... IN (PRWEB) , ... November 24, 2015 , ... The ... newest Special Interest Group (SIG), MultiGP, also known as Multirotor Grand Prix, to represent ... the last few years. Many AMA members have embraced this type of racing and ...
Breaking Biology Technology: