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Msg  44411 of 55120  at  3/17/2012 9:08:44 PM  by

JBWIN


 In response to msg 44410 by  tjmcbntmkr
view thread

Re: A ZFN/piggyBac step closer to autologous liver cell therapy - Look back as far as 2006 ASGCT SGMO PR

 
 Sangamo has dominant ZFP/ZFN IP that will be a factor before commercialization by any organization. You will see ZFP piggybac referenced in a Sangamo PR from 1Jun2006.
 

Sangamo BioSciences to Present Data From Its ZFP Therapeutic(TM) Programs at the 9th Annual Meeting of the American Society of Gene Therapy

 

ZFP Therapeutics Collaborator Chosen for ASGT 'Excellence in Research

Award'


    BALTIMORE, June 1 /PRNewswire-FirstCall/ -- Sangamo BioSciences, Inc.

 (Nasdaq:   SGMO) today announced presentation of data from several of the

 Company's zinc finger DNA-binding protein (ZFP) Therapeutic(TM) programs at

 the 9th Annual Meeting of the American Society of Gene Therapy (ASGT). The

 meeting is being held in Baltimore, Maryland from May 31 through June 4,

 2006.

     Sangamo scientists and their collaborators will make a total of 9

 podium and poster presentations covering several ZFP-mediated gene

 regulation and gene modification therapeutic programs. In addition, Sangamo

 collaborator Angelo Lombardo, from the San Raffaele Telethon Institute for

 Gene Therapy, Milan, Italy, was recognized with an Excellence in Research

 Award for his work described in an abstract entitled, "Towards Gene

 Correction of X-Linked SCID Using Engineered Zinc Finger Nucleases and

 Integrase Defective Lentiviral Delivery".

     "The annual ASGT meeting provides an outstanding forum for sharing the

 latest developments in the field of gene therapy and, as in previous years,

 Sangamo has a significant presence," stated Edward Lanphier, Sangamo's

 president and CEO. "The breadth of material that we are presenting ranging

 from specific therapeutic programs to our internal research programs to

 refine and optimize our technology illustrates the potential broad utility

 of our science."

     Philip Gregory, D.Phil., Vice President, Research at Sangamo noted, "We

 are presenting data for the first time on our process development progress

 in our ZFP Therapeutic program to disrupt the CCR5 gene for the potential

 treatment of HIV/AIDS. In a prize-winning abstract, our collaborators from

 the San Raffaele Telethon Institute in Milan will present data on the

 successful use of non-integrating vectors for delivery of our ZFNs for gene

 correction of X-linked SCID. In addition to updates across our ZFP

 Therapeutic programs in gene regulation, gene correction and gene

 disruption, several of our presentations are focused on technical advances

 and development of our proprietary ZFP nuclease (ZFN(TM)) technology. One

 presentation describes the use of our ZFN technology in targeted

 integration, which we believe has the potential to improve the safety,

 efficiency and utility of gene-based approaches for both therapeutic and

 enabling technology applications."

     Sangamo scientists and their collaborators will make the following

 presentations; the abstract numbers, titles and a brief description of the

 data to be presented are listed below:

     ZFP TF-mediated gene regulation

     -- Abstract #788

        Engineered Zinc Finger Protein Transcription Factors as a Potential

        Therapy for Choriodal Neovascularization

        Saturday, June 3, 2006. Oral Presentation.

        Preclinical animal data will be presented from Sangamo's program to

        develop ZFP TFs for the potential treatment of age-related macular

        degeneration.

     -- Abstract #1010

        Developing a Potential Pain Therapy Using Engineered Zinc Finger

        Protein Repressors of the Sodium Channel PN3

        Saturday, June 3, 2006. Poster Presentation.

        Data will be presented that demonstrate the successful use of ZFP TFs

        to repress the expression of a well-validated pain receptor in primary

        neuronal cells.

 

     ZFN-mediated gene modification

     -- Abstract # 555

        Large-Scale, Flow-Based Electroporation To Deliver Engineered Zinc

        Finger Protein Nucleases That Mediate High-Efficiency Disruption of the

        Human CCR5 Gene

        Friday, June 2, 2006. Poster Presentation.

        Data will be presented demonstrating the efficient delivery of plasmid

        to cells using a GMP compliant flow-based electroporation system.

     -- Abstract # 738

        Towards Gene Correction of X-Linked SCID Using Engineered Zinc Finger

        Nucleases and Integrase Defective Lentiviral Delivery

        Saturday, June 3, 2006. Oral Presentation.

     -- Abstract # 758

        Towards Gene Knock out Therapy for AIDS/HIV: Targeted Disruption of

        CCR5 Using Engineered Zinc Finger Protein Nucleases (ZFNs)

        Saturday, June 3, 2006. Oral Presentation.

     -- Abstract # 787

        Engineered Fok I Heterodimers for Enhanced Zinc Finger Nuclease

        Specificity

        Saturday, June 3, 2006. Oral Presentation.

        Refinements of ZFN engineering will be presented that are designed to

        enhance the specificity of action of ZFNs.

     -- Abstract # 1003

        Targeted Site-Specific Integration in Human Cells Using Designed Zinc

        Finger Nucleases

        Saturday, June 3, 2006. Poster Presentation.

        Data will be presented demonstrating the application of ZFNs for

        efficient targeted integration of DNA sequences.

     -- Abstract # 1040

        Towards Gene Correction Therapy for Wiskott-Aldrich Syndrome Using

        Designed Zinc Finger Endonucleases

        Saturday, June 3, 2006. Poster Presentation.

 

     ZFP Applications

     -- Abstract # 387

        Advantageous Properties of the piggyBac Transposon System for Gene

        Transfer in Human Cells

        Friday, June 2, 2006. Oral Presentation.

 

     Zinc Finger DNA-Binding Proteins

     Zinc Finger DNA-binding Proteins (ZFPs) are a naturally occurring class

 of DNA binding proteins. The DNA recognition and binding function of ZFPs

 can be engineered and thus directed to a targeted sequence of DNA. This

 permits the delivery of a variety of functional domains to a gene-specific

 location. ZFPs are being developed for two significant therapeutic

 applications: gene regulation and gene modification. In the case of

 therapeutic gene regulation, ZFP transcription factors (ZFP TFs) are being

 engineered to either turn on therapeutically beneficial genes or turn off

 the expression of disease-causing genes. For gene modification, ZFPs are

 being used in combination with a DNA cutting enzyme (endonuclease)

 functional domain to generate ZFNs that facilitate the correction of mutant

 gene sequences that cause disease or the disruption of genes that

 facilitate disease progression.

     



 
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