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词条 Protein replacement therapy
释义

  1. See also

  2. References

  3. Further reading

Protein replacement therapy is a medical treatment that supplements or replaces a protein in patients in whom that particular protein is deficient or absent.[1][2] There have been significant advances in this treatment. PRT is being tested in clinical trials with the diseases Progeria and Epidermolysis bullosa dystrophica as a potential treatment. For patients with Epidermolysis bullosa dystrophica there has been promising results. [3][4]

See also

  • Enzyme replacement therapy
  • Gene therapy

References

1. ^{{cite journal | vauthors = Gorzelany JA, de Souza MP | title = Protein replacement therapies for rare diseases: a breeze for regulatory approval? | journal = Science Translational Medicine | volume = 5 | issue = 178 | pages = 178fs10 | date = March 2013 | pmid = 23536010 | doi = 10.1126/scitranslmed.3005007 }}
2. ^{{cite journal | vauthors = Crunkhorn S | title = Regulatory watch: enhanced chance of success for protein replacement therapies | journal = Nature Reviews. Drug Discovery | volume = 12 | issue = 6 | pages = 414 | date = June 2013 | pmid = 23722335 | doi = 10.1038/nrd4027 }}
3. ^{{cite news | first = Colleen | last = Labbe | name-list-format = vanc |url=https://www.niams.nih.gov/newsroom/spotlight-on-research/protein-replacement-therapy-shows-promise|title=Protein Replacement Therapy Shows Promise in Treating Rare Skin Disorder|date=2014-02-05|access-date=2018-10-14 | work = National Institute of Arthritis and Musculoskeletal and Skin Diseases | publisher = U.S. Department of Health and Human Services }}
4. ^{{cite journal | vauthors = Wang X, Ghasri P, Amir M, Hwang B, Hou Y, Khalili M, Khilili M, Lin A, Keene D, Uitto J, Woodley DT, Chen M | title = Topical application of recombinant type VII collagen incorporates into the dermal-epidermal junction and promotes wound closure | journal = Molecular Therapy | volume = 21 | issue = 7 | pages = 1335–44 | date = July 2013 | pmid = 23670575 | pmc = 3704128 | doi = 10.1038/mt.2013.87 | url = http://doi.org/10.1038/mt.2013.87 }}

Further reading

  • {{cite journal | first = Thomas J. | last = Stegmann | name-list-format = vanc | title = Protein Therapy vs. Gene Therapy | url = http://www.drugdiscoverynews.com/index.php?newsarticle=1184 | work = DDNEWS | date = December 2006 }}

1 : Medical treatments

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