Why Only Some Cystic Fibrosis Patients Respond To Treatments That Prevent The Generation Of Truncated Proteins
Omschrijving
Individuals with the genetic lung disorder cystic fibrosis (CF) lack any functional CFTR protein because their genes that encode this protein carry a mutation. One mutation (the W1282X mutation) that results in CF does so because it causes the cellular machinery that converts the initial product of a gene (mRNA) into a functional protein to prematurely stop making CFTR protein. Agents (such as gentamicin) that enable the protein-generating machinery to ignore such mutations have shown benefit in some, but not all, CF patients with the W1282X mutation.
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Categorie: Cystic Fibrosis
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