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:10/27/2015)... , Oct. 27, 2015 Synaptics Inc. (NASDAQ: ... announced that Google has adopted the Synaptics ® ... solutions to power its newest flagship smartphones, the Nexus ... --> --> ... to provide strategic collaboration in the joint development of ...
(Date:10/26/2015)... , Oct. 26, 2015  Delta ID Inc., ... authentication to mobile and PC devices, announced its ActiveIRIS® ... the arrows NX F-02H launched by NTT DOCOMO, INC ... F-02H is the second smartphone to include iris recognition ... in ARROWS NX F-04G in May 2015, world,s first ...
(Date:10/22/2015)... AWRE ), a leading supplier of biometrics software and services, ... 2015.  --> --> Revenue ... of 33% compared to $6.0 million in the same quarter last ... million, or $0.10 per diluted share, which compared to $2.6 million, ... ago.  --> --> Lower ...
Breaking Biology News(10 mins):
(Date:11/26/2015)... November 26, 2015 --> ... specializing in imaging technologies, announced today that it has received ... of the Horizon 2020 European Union Framework Programme for Research ... clinical trial in breast cancer. , --> ... --> --> The study aims ...
(Date:11/25/2015)... -- Studies reveal the differences in species ... the way for more effective treatment for one of the ... --> --> Gum disease ... cats, yet relatively little was understood about the bacteria associated ... conducted by researchers from the WALTHAM Centre for Pet Nutrition ...
(Date:11/25/2015)... ... November 25, 2015 , ... ... of Black Aerospace Professionals (OPBAP) has been formalized with the signing of a ... leaders met with OPBAP leaders Capt. Karl Minter and Capt. Albert Glenn Tuesday, ...
(Date:11/24/2015)... LUMPUR, Malaysia , Nov. 24, 2015 /PRNewswire/ ... global contract research organisation (CRO) market. The trend ... result in lower margins but higher volume share ... increased capacity and scale, however, margins in the ... Research Organisation (CRO) Market ( ), ...
Breaking Biology Technology: