Gene product replacement therapy is an effective strategy when
the deficient gene product is a circulatory peptide or protein.
This forms the standard treatment for insulin dependent
diabetes mellitus, haemophilia and growth hormone
deficiency – conditions that can be treated with systemic
injections. This approach is more difficult when the gene
product is needed for metabolism within specific tissues such as
the central nervous system, where the blood–brain barrier
presents an obstacle to systemic replacement.
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