Lelon R Bulluck Iii lrbulluc at unity.ncsu.edu
Tue Dec 23 10:42:13 EST 1997

Shayne Woodward wrote:
> 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
Cystic fibrosis is caused by a point mutation on Chromosome 4 (I think)
resulting in a disfunctional chlorine channel gene (I'm sure there are
more knowledgeable lurkers who can correct me if I'm wrong).  The
problem with your method of "knocking out" this gene is that it is
already "Knocked-out".  If you sequenced the gene, replaced the faulty
nucleotide, etc., etc, then vectored that engineered gene in, there may
be a chance.  

I'm not up on the current research (as a plant pathology student I
rarely deal with mammalian systems), but I think that such
back-engineering would be ineffective at best.  Cystic Fibrosis causes
systemic problems from the cellular level; in the lungs, kidneys, and
all mucus-producing tissues.  The best approach would be to "fix" the
gene at the un-differentiated embryo stage (and _that's_ a whole other
can o' worms).

I think you are right to worry about ethics here.  This disease is a
_good_ candidate for gene therapy, and I believe such will be attempted
in our life-time.  Finally, I was just opening dialog. . . not starting
a war.  So please be kind. . .I'm new at this.

Later, Russ

Russ Bulluck
Graduate Student
Plant Pathology
Box 7616
Raleigh, NC  27695
Sometimes you have to look reality in the eye and deny it.
	--Garrison Keillor
Never be afraid to sit awhile and think.
	--Lorraine Hansberry (A Raisin in the Sun)

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