FRINK Linked Data Fragments server

Matches in SemOpenAlex for { <https://semopenalex.org/work/W1427384429> ?p ?o ?g. }

• W1427384429 abstract "Publisher Summary Progress on understanding the molecular basis of cystic fibrosis (CF) has been extremely encouraging since the gene was identified. This chapter summarizes the new treatments of CF that are beginning to emerge and also discusses several more traditional approaches that are also showing signs of promise. CF is the most common lethal, recessive genetic disease of the Caucasian population. The disease is manifest in many secretory epithelial surfaces of the body. Pharmacological approaches to CF are least impacted by the knowledge of CFTR and its associated biochemical functions. The delivery of wild type protein to a patient lacking the associated biochemical activity is the present treatment of choice for many protein deficiency diseases, especially genetic diseases. Such treatment directly augments the missing activity. Proteins purified from human sources (such as blood or placenta) and more recently produced by recombinant means, have been successfully used. The fusion of CFTR from its delivery vehicle into the membrane of recipient cells is likely to be an inefficient process. The next significant milestone is to establish the feasibility of transfer of partially purified CFTR into recipient cells from a CF patient followed by the measurements of membrane function. Such an experiment will establish the feasibility of protein replacement therapy. The concept of gene replacement therapy is also very simple; a preparation of CFTR coding sequences in some suitable vector in a viral or other carrier delivered directly to the airway cells of CF patients. Defective adenoviruses, at present, appear a potentially very promising approach to CF gene therapy. Approaches to therapy based on the knowledge of the gene have the advantage of being completely rational, and there is a high expectation that they can eventually be efficacious." @default.
• W1427384429 created "2016-06-24" @default.
• W1427384429 creator A5055235682 @default.
• W1427384429 date "1992-01-01" @default.
• W1427384429 modified "2023-09-25" @default.
• W1427384429 title "Chapter 25. Emerging Therapies for Cystic Fibrosis" @default.
• W1427384429 cites W126651838 @default.
• W1427384429 cites W1513412619 @default.
• W1427384429 cites W1513817400 @default.
• W1427384429 cites W1534650679 @default.
• W1427384429 cites W1537652082 @default.
• W1427384429 cites W1553673732 @default.
• W1427384429 cites W1786021994 @default.
• W1427384429 cites W1893545268 @default.
• W1427384429 cites W1965016620 @default.
• W1427384429 cites W1969697265 @default.
• W1427384429 cites W1975971473 @default.
• W1427384429 cites W1979040675 @default.
• W1427384429 cites W1979487106 @default.
• W1427384429 cites W1982071077 @default.
• W1427384429 cites W1984729482 @default.
• W1427384429 cites W1987325291 @default.
• W1427384429 cites W1987600685 @default.
• W1427384429 cites W1988101098 @default.
• W1427384429 cites W1989243981 @default.
• W1427384429 cites W1990637933 @default.
• W1427384429 cites W1991148544 @default.
• W1427384429 cites W1995240556 @default.
• W1427384429 cites W1997182007 @default.
• W1427384429 cites W1998130586 @default.
• W1427384429 cites W1999495254 @default.
• W1427384429 cites W2009482334 @default.
• W1427384429 cites W2010309531 @default.
• W1427384429 cites W2010691166 @default.
• W1427384429 cites W2014495927 @default.
• W1427384429 cites W2015881103 @default.
• W1427384429 cites W2016532191 @default.
• W1427384429 cites W2031860235 @default.
• W1427384429 cites W2032510845 @default.
• W1427384429 cites W2032940647 @default.
• W1427384429 cites W2035177679 @default.
• W1427384429 cites W2035714544 @default.
• W1427384429 cites W2036774130 @default.
• W1427384429 cites W2039599894 @default.
• W1427384429 cites W2046069397 @default.
• W1427384429 cites W2047930213 @default.
• W1427384429 cites W2051287901 @default.
• W1427384429 cites W2058253654 @default.
• W1427384429 cites W2061429557 @default.
• W1427384429 cites W2062120260 @default.
• W1427384429 cites W2068217887 @default.
• W1427384429 cites W2071977262 @default.
• W1427384429 cites W2075257287 @default.
• W1427384429 cites W2076091117 @default.
• W1427384429 cites W2076515495 @default.
• W1427384429 cites W2078379563 @default.
• W1427384429 cites W2078380427 @default.
• W1427384429 cites W2080423498 @default.
• W1427384429 cites W2100432609 @default.
• W1427384429 cites W2102886871 @default.
• W1427384429 cites W2107312415 @default.
• W1427384429 cites W2120337235 @default.
• W1427384429 cites W2121576595 @default.
• W1427384429 cites W2123356870 @default.
• W1427384429 cites W2124951171 @default.
• W1427384429 cites W2125391335 @default.
• W1427384429 cites W2125428336 @default.
• W1427384429 cites W2129859001 @default.
• W1427384429 cites W2142285863 @default.
• W1427384429 cites W2144248619 @default.
• W1427384429 cites W2160327135 @default.
• W1427384429 cites W2169715286 @default.
• W1427384429 cites W2199020419 @default.
• W1427384429 cites W2212152199 @default.
• W1427384429 cites W2317516580 @default.
• W1427384429 cites W2322128806 @default.
• W1427384429 cites W2338243080 @default.
• W1427384429 cites W2407398125 @default.
• W1427384429 cites W2410214285 @default.
• W1427384429 cites W44006843 @default.
• W1427384429 doi "https://doi.org/10.1016/s0065-7743(08)60423-6" @default.
• W1427384429 hasPublicationYear "1992" @default.
• W1427384429 type Work @default.
• W1427384429 sameAs 1427384429 @default.
• W1427384429 citedByCount "0" @default.
• W1427384429 crossrefType "book-chapter" @default.
• W1427384429 hasAuthorship W1427384429A5055235682 @default.
• W1427384429 hasConcept C104317684 @default.
• W1427384429 hasConcept C111599444 @default.
• W1427384429 hasConcept C135983454 @default.
• W1427384429 hasConcept C142724271 @default.
• W1427384429 hasConcept C203014093 @default.
• W1427384429 hasConcept C2776938444 @default.
• W1427384429 hasConcept C2779134260 @default.
• W1427384429 hasConcept C2908647359 @default.
• W1427384429 hasConcept C54355233 @default.
• W1427384429 hasConcept C60644358 @default.
• W1427384429 hasConcept C70721500 @default.
• W1427384429 hasConcept C71924100 @default.
• W1427384429 hasConcept C86803240 @default.

• next