Mode of action of RNA/DNA oligonucleotides: Progress in the development of gene repair as a therapy for α1-antitrypsin deficiency

Chest

Volumn 121, Issue 3, 2002, Pages 91S-97S

  Metz, Richard ^a   DiCola, Mike ^a   Kurihara, Takao ^a   Bailey, Austin ^a   Frank, Bruce ^a   Roecklein, Bryan ^a   Blaese, Michael ^a  

^a NONE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

DNA; OLIGONUCLEOTIDE; PROTEIN; PROTEIN MUTS; RECA PROTEIN; RNA; UNCLASSIFIED DRUG;

ALPHA 1 ANTITRYPSIN DEFICIENCY; CHROMOSOME; CONFERENCE PAPER; CONTROLLED STUDY; DNA REPAIR; DNA STRUCTURE; EPISOME; ESCHERICHIA COLI; EUKARYOTIC CELL; GENE CONVERSION; NONHUMAN; PRIORITY JOURNAL; PROKARYOSIS; PROTEIN FUNCTION; RNA STABILITY; RNA STRUCTURE;

ESCHERICHIA COLI;

EID: 0036129569     PISSN: 00123692     EISSN: None     Source Type: Journal    
DOI: None     Document Type: Conference Paper

References (24)

1. Alpha-1 antitrypsin deficiency
2. A-1-Antitrypsin deficiency
3. Alpha 1-Antitrypsin: Molecular pathology, leu-kocytes, and tissue damage
4. Longitudinal follow-up of patients with α1-protease inhibitor deficiency before and during therapy with IV 31-protease inhibitor
5. Sustained secretion of human alpha-1-antitrypsin from murine muscle transduced with adeno-associated cirus vectors
6. Gene repair using chimeric RNA/DNA oligonucleotides
7. Therapeutic gene targeting
8. Targeted gene correction of episomal DNA in mammalian cells mediated by a chimeric RNA/DNA oligonucleotide
9. Correction of the mutation responsible for sickle cell anemia by an RNA-DNA oligonucleotide
10. Targeted nucleotide exchange in the alkaline phosphatase gene of HuH-7 cells mediated by a chimeric RNA/DNA oligonucleotide
11. In vivo site-directed mutagenesis of the factor IX gene by chimeric RNA/DNA oligonucleotides
12. Correction of the UDP-glucuronosyltransferase gene defect in the Gunn rat model of Crigler-Najjar syndrome type I with a chimeric oligonucleotide
13. Localized in vivo genotypic and phenotypic correction of the albino mutation in skin by RNA-DNA oligonucleotide
14. Rescue of dystrophin expression in mdx mouse muscle by RNA/DNA oligonucleotides
15. In vivo targeted repair of a point mutation in the canine dystrophin gene by a chimeric RNA/DNA oligonucleotide
16. Gene correction of the apolipoprotein (Apo) E2 phenotype to wild-type ApoE3 by in situ chimeraplasty
17. A plausible mechanism for gene correction by chimeric oligonucleotides
18. Persistent failures in gene repair
19. Stable and inheritable changes in genotype and phenotype of albino melanocytes induced by an RNA-DNA oligonucleotide
20. Primer-directed enzymatic amplification of DNA with a thermostable DNA polymerase
21. Improved M13 phage cloning vectors and host strains: Nucleotide sequences of the M13mp18 and pUC19 vectors
22. High levels of foreign gene expression in hepatocytes after tail vein injections of naked plasmid DNA