Publication Date:
1996-09-06
Description:
A chimeric oligonucleotide composed of DNA and modified RNA residues was used to direct correction of the mutation in the hemoglobin betaS allele. After introduction of the chimeric molecule into lymphoblastoid cells homozygous for the betaS mutation, there was a detectable level of gene conversion of the mutant allele to the normal sequence. The efficient and specific conversion directed by chimeric molecules may hold promise as a therapeutic method for the treatment of genetic diseases.〈br /〉〈span class="detail_caption"〉Notes: 〈/span〉Cole-Strauss, A -- Yoon, K -- Xiang, Y -- Byrne, B C -- Rice, M C -- Gryn, J -- Holloman, W K -- Kmiec, E B -- New York, N.Y. -- Science. 1996 Sep 6;273(5280):1386-9.〈br /〉〈span class="detail_caption"〉Author address: 〈/span〉Department of Pharmacology, Kimmel Cancer Center, Thomas Jefferson University, 233 South 10th Street, Philadelphia, PA 19107, USA.〈br /〉〈span class="detail_caption"〉Record origin:〈/span〉 〈a href="http://www.ncbi.nlm.nih.gov/pubmed/8703073" target="_blank"〉PubMed〈/a〉
Keywords:
Alleles
;
Anemia, Sickle Cell/*genetics/therapy
;
Base Sequence
;
Cells, Cultured
;
*Gene Conversion
;
Genetic Therapy
;
Globins/genetics
;
Hemoglobin, Sickle/*genetics
;
Humans
;
Lymphocytes
;
Molecular Sequence Data
;
Oligodeoxyribonucleotides/*genetics
;
Oligoribonucleotides/*genetics
;
Point Mutation
;
Polymerase Chain Reaction
;
Polymorphism, Restriction Fragment Length
;
*Transfection
Print ISSN:
0036-8075
Electronic ISSN:
1095-9203
Topics:
Biology
,
Chemistry and Pharmacology
,
Computer Science
,
Medicine
,
Natural Sciences in General
,
Physics
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