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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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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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