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Genetic therapy for congenital myopathies
Current Opinion in Neurology, 2021Purpose of review There has been an explosion of advancement in the field of genetic therapies. The first gene-based treatments are now in clinical practice, with several additional therapeutic programs in various stages of development.
Nika, Maani +2 more
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Genetic therapies in cystic fibrosis
Current Opinion in Pulmonary Medicine, 2023Purpose of review Advances in cystic fibrosis (CF) therapies over the past decade pivotally changed the morbidity and mortality of CF with the advent of cystic fibrosis transmembrane conductance regulator (CFTR) modulators that rescue dysfunctional CFTR protein in individuals with eligible genotypes.
Jennifer L, Taylor-Cousar +3 more
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Clinical Trials of Genetic Therapy with Antisense DNA and DNA Vectors
, 2020A Brief History of Genetic Therapy: Gene Therapy, Antisense Technology, and Genomics, James W. Hawkins Preclinical Development of Oligonucleotide Therapeutics for Cancer: Regulatory Aspects, Chang-Ho Ahn and Joseph J.
E. Wickstrom
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Transplantation Proceedings, 2005
Cellular genetic therapy is the ultimate frontier for those pathologies that are consequent to a specific nonfunctional cellular type. A viable cure for there kinds of diseases is the replacement of sick cells with healthy ones, which can be obtained from the same patient or a different donor.
Del Vecchio, F +4 more
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Cellular genetic therapy is the ultimate frontier for those pathologies that are consequent to a specific nonfunctional cellular type. A viable cure for there kinds of diseases is the replacement of sick cells with healthy ones, which can be obtained from the same patient or a different donor.
Del Vecchio, F +4 more
openaire +4 more sources
Genetic therapies and xenotransplantation
Expert Opinion on Biological Therapy, 2002The number of patients in need of an organ transplant is increasing, while the number of satisfactory sources of organs has declined in many countries [101]. The resulting shortage of human organs has spurred an urgent effort to investigate alternative therapies, including the use of animal organs, tissues and cells (i.e., xenotransplantation ...
Brenda M, Ogle, Jeffrey L, Platt
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Programming hMSCs into Potential Genetic Therapy in Cancer.
Critical Reviews in Eukaryotic Gene Expression, 2019Based on their distinct characteristics, such as self-renewal and differentiation potential, human mesenchymal stem cells (hMSCs) have been proposed as a feasible tool for cancer therapy.
N. Serakinci, Huseyin Cagsin
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Gene Therapy for Genetic Diseases
Cancer Investigation, 1989(1989). Gene Therapy for Genetic Diseases. Cancer Investigation: Vol. 7, No. 2, pp. 179-192.
D B, Kohn, W F, Anderson, R M, Blaese
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Genetic prodrug activation therapy
The Lancet, 1997Genetic prodrug activation therapy shows promise as a therapeutic option for the treatment of cancer as well as a variety of other diseases. It involves the insertion of a gene encoding a drug-metabolizing enzyme into cells and the systemic administration of a prodrug. The prodrug is converted to a cytotoxic agent by the action of the expressed enzyme.
L A, Martin +4 more
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