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Research in HIV via CCR5 Delta 32

May 17, 2009

Hello Dr Franscino, I am curious if the following is currently being researched and if so how I could be involved. I know that scientists have recently been able to induce differentiated skin cells into a stem cell-like state using 4 genes and more recently 4 proteins. My thought is, if this proves to be true would it be possible to take skin cells from an HIV+ individual, dedifferentiate to the stem cell like phase and then introduce the specific mutation to the CCR5 Delta 32 gene mutation which confers HIV resistance by altering the receptor HIV uses to enter the human body? Then the stem cells could be driven to develop to the bone marrow stem cells and a bone marrow transplant could be performed. Since the new cells would have the same MHC complexes as the individual, would this reduce the risks of a bone marrow transplant and confer resistance to HIV?

I am aware that there would be a number of obstacles to overcome, ie ensuring the mutation enters the gene in the correct place and only at that specific area, but it seems to me like this would be an exciting therapy to treat HIV. Would it be possible to modify the integrase virus use so it would only recognize a specific DNA sequence and insert the desired mutation appropriately?

The funny thing is, this idea just came to me last night (I'm applying to MD school this summer and just finishing up Bio 102) while watching a program on Discovery where they discussed the CCR5 Delta 32 mutation. I got to thinking, and this is it.

If nobody is currently researching this potential therapy, would you mind telling me if I could help get it started? I probably can't because I'm an undergrad and haven't even been accepted into medical school yet, but could you imagine if this could work? All the lives that could be saved.



Response from Dr. Frascino

Hi Patrick,

No, your concept is not currently being studied and consequently no, you can not be involved. Your hypothesis is faulty on many immunological and genetic levels. You'll understand this more completely after you've been through medical school.

Certainly more appropriate CCR5-delta 32 gene mutation research is ongoing. Good luck with your medical school applications.

Dr. Bob

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