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Biotech / Medical : Targeted Gene Repair
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To: John McCarthy who started this subject9/15/2002 8:25:49 PM
From: John McCarthy   of 22
 
TARGETED GENE THERAPY

Subject:SSO - (Single-stranded oligonucleotide)
(The following material is presented in Ascending Date Order)

This post is generated by a program that reads a database and generates this file. Errors will result from incorrect database material. Updates to the database automatically reflected in this file.

Researcher Name shown is my estimate of Principal Researcher

DB Key:1999-SSO-A
Doc:Abstract
Vector:SSO Single-stranded oligonucleotide
Experiment:Failed or Not Applicable
Of Note:A sequence-specific genomic delivery system for the correction of chromosomal mutations was designed by incorporating two different binding domains into a single-stranded oligonucleotide. A repair domain (RD) contained the native sequence of the target region. A third strand-forming domain (TFD) was designed to form a triplex by Hoogsteen interactions. The design was based upon the premise that the RD will rapidly form a heteroduplex that is anchored synergistically by the TFD.

10/17/1999-----Culver KW-----Novartis Pharmaceuticals Corp
Title:Correction of chromosomal point mutations in human cells with bifunctional oligonucleotides
Link:http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?cmd=Retrieve&db=PubMed&list_uids=10504700&dopt=Abstract

Snippet:
Transfection of ADA-deficient human lymphocytes corrected the mutant sequence in 1-2% of cells. Neither the RD or TFD individually corrected the mutation. Transfection of p53 mutant human glioblastoma cells corrected the mutation and induced apoptosis in 7.5% of cells.
Transfection of ADA-deficient human lymphocytes corrected the mutant sequence in 1-2% of cells. Neither the RD or TFD individually corrected the mutation. Transfection of p53 mutant human glioblastoma cells corrected the mutation and induced apoptosis in 7.5% of cells.
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