TARGETED GENE THERAPY
Subject:Duchenne Muscular Dystrophy (DMD)
(The following material is presented in Ascending Date Order)
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Researcher Name shown is my estimate of Principal Researcher
DB Key:2000-DMD-B
Doc:Abstract
Vector:RDO RNA-DNA chimeric oligonucleotide
Experiment:Successful
Of Note:Muscular Dystrophy
5/1/2000-----Rando, Thomas M.D., Ph.D.-----Department of Neurology and Neurological Sciences, Palo Alto Veterans Affairs Medical Center and Stanford University School of Medicine, Palo Alto, CA 94305, USA.
Title:Rescue of dystrophin expression in mdx mouse muscle by RNA/DNA oligonucleotides
Link:http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?cmd=Retrieve&db=PubMed&list_uids=10805797&dopt=Abstract
Disease Information:http://www.mdausa.org/disease/dmd.html
Snippet:
Ten weeks after single injections, the range of the number of dystrophin-positive fibers was similar to that seen after 2 wk, suggesting that the expression was stable, as would be predicted for a gene-conversion event
DB Key:2000-DMD-A
Doc:NINDS web site
Vector:RDO RNA-DNA chimeric oligonucleotide
Experiment:Failed or Not Applicable
Of Note:None
5/1/2000-----Various DMD researchers-----Natcher Conference Center, NIH, Bethesda, MD
Title:Workshop on Therapeutic Approaches for Duchenne Muscular Dystrophy
Link:http://accessible.ninds.nih.gov/news_and_events/dmdmtngsummary.htm
Disease Information:http://www.mdausa.org/disease/dmd.html
DB Key:2000-DMD-F
Doc:Genome Network News
Vector:RDO RNA-DNA chimeric oligonucleotide
Experiment:Successful
Of Note:First Dog - Point Mutation
6/3/2000-----Bartlett RJ-----National Institute of Arthritis and Musculoskeletal and Skin Diseases
Title:Genetic surgery for muscular dystrophy in golden retrievers
Link:http://www.genomenewsnetwork.com/articles/06_00/muscular_dystrophy.shtml
Disease Information:http://www.mdausa.org/disease/dmd.html
Snippet:
One major problem is delivering enough of the oligonucleotide to all affected muscles
DB Key:2000-DMD-J
Doc:Abstract
Vector:RDO RNA-DNA chimeric oligonucleotide
Experiment:Successful
Of Note:First Dog - Point Mutation
6/18/2000-----Bartlett RJ-----Department of Veterinary Medicine and Surgery
Title:In vivo targeted repair of a point mutation in the canine dystrophin gene by a chimeric RNA/DNA oligonucleotide
Link:http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?cmd=Retrieve&db=pubmed&dopt=Abstract&list_uids=10835598
2nd Link:http://www.genomenewsnetwork.com/articles/06_00/muscular_dystrophy.shtml
Disease Information:http://www.mdausa.org/disease/dmd.html
Snippet:
Reverse transcription-polymerase chain reaction (RT-PCR) analysis of exons 5-10 demonstrated increasing levels of exon 7 inclusion with time. An isolated exon 7-specific dystrophin antibody confirmed synthesis of normal-sized dystrophin product and positive localization to the sarcolemma. Chromosomal repair in muscle tissue was confirmed by restriction fragment length polymorphism (RFLP)-PCR and sequencing the PCR product. This work provides evidence for the long-term repair of a specific dystrophin point mutation in muscle of a live animal using a chimeric oligonucleotide
DB Key:2001-DMD-F
Doc:Biography
Vector:SFHR Small Fragment DNA
Experiment:Failed or Not Applicable
Of Note:SFHR
4/1/2001-----Kapsa R-----Melbourne Neuromuscular Research Institute
Title:Dr. Robert Kapsa
Link:http://www.mda.org.au/research/nrc_kapsa.html
Disease Information:http://www.mdausa.org/disease/dmd.html
Snippet:
the lipofection used here appeared to have adverse effects on subsequent cell viability and corrected cells did not express dystrophin transcript
DB Key:2001-DMD-A5
Doc:Abstract
Vector:SFHR Small Fragment DNA
Experiment:Failed or Not Applicable
Of Note:SFHR
4/1/2001-----Kapsa R-----Melbourne Neuromuscular Research Institute
Title:In vivo and in vitro correction of the mdx dystrophin gene nonsense mutation by short-fragment homologous replacement
Link:http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?cmd=Retrieve&db=PubMed&list_uids=11426463&dopt=Abstract
Disease Information:http://www.mdausa.org/disease/dmd.html
Snippet:
the lipofection used here appeared to have adverse effects on subsequent cell viability and corrected cells did not express dystrophin transcript
DB Key:2002-DMD-A4
Doc:Abstract
Vector:RDO RNA-DNA chimeric oligonucleotide
Experiment:Successful
Of Note:None
4/1/2002-----Rando, Thomas M.D., Ph.D.-----Department of Neurology and Neurological Sciences, Stanford University School of Medicine, Stanford, CA 94305.
Department of Neurology and Neurological Sciences, Stanford University School of Medicine, Stanford, CA 94305.
Title:Dystrophin Gene Repair in mdx Muscle Precursor Cells In Vitro and In Vivo Mediated by RNA-DNA Chimeric Oligonucleotides.
Dystrophin Gene Repair in mdx Muscle Precursor Cells In Vitro and In Vivo Mediated by RNA-DNA Chimeric Oligonucleotides.
Link:http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?cmd=Retrieve&db=PubMed&list_uids=11936970&dopt=Abstract
Disease Information:http://www.mdausa.org/disease/dmd.html
Snippet:
When muscle precursor cells were subsequently derived from muscles injected with a targeting chimeraplast, we found that gene repair had occurred in these cells as well
DB Key:2002-DMD-A6
Doc:Abstract
Vector:SFHR Small Fragment DNA
Experiment:Failed or Not Applicable
Of Note:SFHR
6/9/2002-----Kapsa R-----Melbourne Neuromuscular Research Institute
Title:Targeted gene correction in the mdx mouse using short DNA fragments: towards application with bone marrow-derived cells for autologous remodeling of dystrophic muscle
Link:http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?cmd=Retrieve&db=PubMed&list_uids=12032690&dopt=Abstract
Disease Information:http://www.mdausa.org/disease/dmd.html
Snippet:
Using wt bone marrow as a model, this study investigates systemic application of bone marrow-derived cells as potential vehicles to deliver corrected (ie wt) dys locus to dystrophic muscle. |
