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Biotech / Medical : Targeted Gene Repair

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To: John McCarthy who started this subject	9/15/2002 8:22:09 PM
From: John McCarthy	of 22

TARGETED GENE THERAPY Subject:Hemophilia (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-HEMO-A* Doc:Web Page Article Vector:RDO RNA-DNA chimeric oligonucleotide Experiment:Failed or Not Applicable Of Note:None 1/1/1999-----Baxter Health Care-----Baxter Health Care Title:Types of Vectors Link:http://www.hemophiliagalaxy.com/1_PATIENTS/Q_A/future/directory_topics/SEC_03d/ Disease Information:http://www.icondata.com/health/pedbase/files/HEMOPHI2.HTM Snippet: Describes the 7 Vector Types *DB Key:1999-HEMO-B* Doc:Abstract Vector:RDO RNA-DNA chimeric oligonucleotide Experiment:Failed or Not Applicable Of Note:FACTOR IX 4/15/1999-----Steer CJ-----University of Minnesota Title:Nucleotide exchange in genomic DNA of rat hepatocytes using RNA/DNA oligonucleotides. Targeted delivery of liposomes and polyethyleneimine to the asialoglycoprotein receptor Link:http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?cmd=Retrieve&db=PubMed&list_uids=10187800&dopt=Abstract Disease Information:http://www.icondata.com/health/pedbase/files/HEMOPHI2.HTM Snippet: The oligonucleotides were encapsulated in positive, neutral, and negatively charged liposomes containing galactocerebroside or complexed with lactosylated polyethyleneimine. The formulations were evaluated for stability and efficiency in targeting hepatocytes via the asialoglycoprotein receptor. Physical characterization and electron microscopy revealed that the oligonucleotides were efficiently encapsulated within the liposomes, with the positive and negative formulations remaining stable for at least 1 month *DB Key:2000-HEMO-A* Doc:Web Page Article Vector:RDO RNA-DNA chimeric oligonucleotide Experiment:Failed or Not Applicable Of Note:None 1/1/2000-----Lillicrap,David Dr.-----Queen's University in Kingston Title:Evaluation of a model system for a site-specific correction of hemophilic mutations Link:http://www.hemophilia.ca/en/3.1.1.html#8 Disease Information:http://www.icondata.com/health/pedbase/files/HEMOPHI2.HTM Snippet: On Brian's return to Kingston, despite incorporating new aspects of Dr. Kmiec's methodology, we continued to experience very poor levels of genetic alteration *DB Key:2001-HEMO-D* Doc:Abstract Vector:RDO RNA-DNA chimeric oligonucleotide Experiment:Successful Of Note:beta-Thalassemia 3/1/2001-----Li ZH-----National Laboratory of Medical Molecular Biology, Institute of Basic Medical Sciences, Beijing, 100005, China Title:Targeted correction of the point mutations of beta-thalassemia and targeted mutagenesis of the nucleotide associated with HPFH by RNA/DNA oligonucleotides Link:http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?cmd=Retrieve&db=PubMed&list_uids=11500064&dopt=Abstract 2nd Link:http://www.icondata.com/health/pedbase/files/THALASSE.HTM Disease Information:http://www.icondata.com/health/pedbase/files/THALASSE.HTM Snippet: successfully corrected the point mutation of the betaE gene with the highest correction efficiency of 1.9%

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