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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 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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