I am a third year biology student, and I have an interest in biological
vectors. Please excuse my ignorance if I may seem "naive"
knowledge-wise, but here it is. Cystic fibrosis is caused by a
single-gene mutation, correct? Now, theoretically, would it be
possible to synthesize an appropriate vector to "knock out" this
dysfunctional gene in CF patients? I have done some research on the
uses of these vectors, and I am not sure about this but am assuming
that since this technique of using vectors and "knock out" procedures
have been developed, it has been a possibility that single gene
mutations could be rendered nonfunctional, thus presenting a "cure" for
CF and other single-gene mutation diseases. Now, theory aside, the
technical aspect. Is this technique being implemented presently? From
what I can see, some major technical barriers would be making this
vector tissue-specific. Even if this technique did become perfected,
would it even be used due to ethics, ie. "playing God", tampering with
a human genome? Is this a plausible theory, or am I just watching too
much Star Trek?
Shayne
