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pro vyhledávání: '"Randy C. Eisensmith"'
Autor:
Randy C. Eisensmith, Savio L. C. Woo
Publikováno v:
Gene Therapy for Diseases of the Lung ISBN: 9781003064619
Gene Therapy for Diseases of the Lung
Gene Therapy for Diseases of the Lung
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::627f72b84c42877a7f5c48054c0e7ebc
https://doi.org/10.1201/9781003064619-17
https://doi.org/10.1201/9781003064619-17
Publikováno v:
The Journal of Gene Medicine. 3:51-58
Background A major limitation of adenovirus-mediated gene therapy for metabolic and inherited diseases is the instability of transgene expression in vivo. This instability results, at least in part, from the inability of the vector genome to maintain
Publikováno v:
Mental Retardation and Developmental Disabilities Research Reviews. 5:136-143
Gene therapy is being explored as a means of treating a variety of genetic diseases. Because of the central role of the liver in many metabolic pathways, and the relative ease with which foreign genes can be delivered to hepatocytes, monogenic disord
Publikováno v:
Mental Retardation and Developmental Disabilities Research Reviews. 5:113-116
Phenylketonuria (PKU) is a recessive trait that usually produces a clear-cut Mendelian pattern in individual families. However, the distribution of phenotypes among families is heterogeneous in severity and response to dietary treatment, spanning fro
Publikováno v:
Molecular Endocrinology. 11:833-837
Type 1 diabetes mellitus is caused by a lack of insulin that results from the autoimmune destruction of the pancreatic beta-cells. Severe diabetes, if not controlled by periodic insulin injections, can lead to ketoacidosis and death. We have previous
Autor:
Savio L. C. Woo, Randy C. Eisensmith
Publikováno v:
Thrombosis and Haemostasis. 78:024-030
Over the past five years, significant advances have been made in the development of novel viral vector systems for the treatment of hemophilia B by somatic gene therapy. At present, both a sustained but partial or a complete but transient correction
Publikováno v:
Proceedings of the National Academy of Sciences. 93:14804-14808
The ob / ob mouse is genetically deficient in leptin and exhibits both an obese and a mild non-insulin-dependent diabetic phenotype. To test the hypothesis that correction of the obese phenotype by leptin gene therapy will lead to the spontaneous cor
Autor:
Savio L. C. Woo, Randy C. Eisensmith
Publikováno v:
Journal of Inherited Metabolic Disease. 19:412-423
Gene therapy is the delivery of genetic material to specific cell types of an organism to alter its physiology or function. This technology is being explored as a means of treating diseases caused by deficiencies of hepatic gene products. The two dis
Autor:
Mark A. Kay, Charles N. Landen, H. Wang, Dwight A. Bellinger, R. E. Cross, Kenneth M. Brinkhous, B. Fang, Savio L. C. Woo, Marjorie S. Read, G. Gordon, Randy C. Eisensmith, Ping-Chuan Hu
Publikováno v:
Human Gene Therapy. 6:1039-1044
Hemophilia B is caused by a deficiency of blood clotting factor IX (FIX). Previous studies have shown that the delivery of a recombinant adenoviral vector expressing canine FIX (cFIX) resulted in a complete correction of hemophilia B in FIX-deficient
Complete Spectrum of PAH Mutations in Tataria: Presence of Slavic, Turkic and Scandinavian Mutations
Autor:
Alexei A. Goltsov, Alexei I. Kuzmin, Randy C. Eisensmith, Eugene I. Schwartz, Savio L. C. Woo, Naila A. Sergeeva
Publikováno v:
European Journal of Human Genetics. 3:246-255
Phenylketonuria (PKU) is an autosomal recessive disorder associated with a deficiency of hepatic phenylalanine hydroxylase (PAH). Although the molecular lesions present in the PAH genes of PKU patients have previously been determined in several Slavi