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A Novel Adeno-Associated Virus for Gene Therapy

20:00 EDT 2 Aug 2015 | NIH

Scientists at the NIH disclosed a novel adeno-associated virus (AAV) termed "44-9." AAV44-9 based vectors have high gene transfer activity in a number of cell types, including salivary gland cells, liver cells, and different types of neurons (e.g., cells of the cortex, olfactory bulb, and brain stem, and Purkinje cells of the cerebellum). These vectors can increase the transduction efficiency and decrease the potential of being neutralized by preexisting antibodies compared to the wild type AAV. Preliminary results from animal studies suggest that AAV44-9 vectors can efficiently deliver genes of interest, and the protein products of the delivered genes can be detected in bloodstream and at the local tissues. Therefore, these vectors are suitable for gene therapy for cells/tissues that are not efficiently targeted by other vectors.

IC: 
NHLBI
NIH Ref. No.: 
E-175-2015-0
TAB No: 
TAB-2954
Advantages: 
  • High gene transfer activity in a number of cell types including salivary gland cells, liver cells, and different types of neurons (e.g., cells of the cortex, olfactory bulb, and brain stem, and Purkinje cells of the cerebellum).
  • As a gene transfer vector for cells that are not efficiently targeted by other vector.
Applications: 
  • AAV44-9 can be used as a delivery vector in gene therapy.
Development Status: 
  • In vitro data available
  • In vivo data available (animal)
Provider Technology ID: 
2954
Updated On: 
May 1, 2019
Date Published: 
Monday, August 3, 2015
Patent Application: 
62/160,552
Patent Authority: 
US
Lead Inventor: 
Inventor IC: 
NHLBI
NIDCR
Inventor Lab URL: 
https://irp.nih.gov/pi/john-chiorini
LPM FIrst Name: 
Vladimir
LPM Last Name: 
Knezevic
LPM Address: 
BG 12A RM 3011 12 SOUTH DR
LPM City: 
Bethesda
Inv Is lead: 
LPM Zip: 
20892
LPM State: 
MD
LPM Phone: 
301.443.5560
LPM Suffix: 
M.D.
LPM Organization: 
NIH Office of Technology Transfer
DTDT Classification: 
Arthritis
Cancer
Central Nervous System
Diabetes
Endocrine
Therapeutic (biologic)
Gene-based
In vitro data available
In vivo data available (animal)
DTDT Description: 
Arthritis
Cancer
Central Nervous System
Diabetes
Endocrine
Therapeutic (biologic)
Gene-based
In vitro data available
In vivo data available (animal)
Pat Filing Date: 
2015-05-12
Publication Link: 
https://www.ncbi.nlm.nih.gov/pubmed/16641301
Publication Caption: 
PMID 16641301
Publication Title: 

Schmidt M, et al.

Application Term: 
Technology Term: 
Collaboration Sought: 
Yes
Institute or Center: 
Collaboration Opportunity: 

The National Institute of Dental and Craniofacial Research is seeking statements of capability or interest from parties interested in collaborative research to further develop, evaluate or commercialize AAV44-9 vector for gene therapy. For collaboration opportunities, please contact David Bradley, Ph.D. at bradleyda@nidcr.nih.gov.

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Related Invention: 
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E Number Only: 
E-175-2015
Inventor First Name: 
John
Giovanni
Inventor Last Name: 
Chiorini
Di Pasquale

Original Article: A Novel Adeno-Associated Virus for Gene Therapy

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