Re: mito article

[Guest guest]

Interesting article. I think it is still in the early theoretical

stages. I did find several other articles in PubMed, co-authored by

Weissig. Here is one recent reference.

http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?

cmd=Retrieve&db=pubmed&dopt=Abstract&list_uids=15384945

>

>

> I just found this article while doing a search today. It's  from

> 2001. Has

> anyone heard anymore about this?

>

> Antiseptic May Target Mitochondrial Disease

> Certain mitochondrial diseases are currently beyond the reach of gene 

> therapy, but a chemical commonly used as an antiseptic might be the

> magic bullet 

> for that problem.   

>

> A DQAsome is a hollow capsule formed from many  bolaform-shaped DQA

> molecules

> (highlighted). The capsule might make an  ideal gene therapy vector

> for

> mitochondrial diseases. 

> Gene therapy — the delivery of corrective genes to make up for a

> genetic 

> defect — holds promise for treating most genetic diseases, and in

> some cases, is 

> already being tested in clinical trials. But some mitochondrial

> diseases are 

> off-limits to current gene therapy techniques because they're caused

> by

> defects  in hard-to-reach genes.

> Most of our genetic material (DNA) is housed within the nuclei, the

> control 

> centers found in nearly all our cells. But some DNA is housed in the 

> mitochondria, the tiny powerhouses that provide energy to cells.

> Defects in 

> mitochondrial DNA can cause energy deficits that lead to the extreme

> fatigue and 

> weakness characteristic of mitochondrial muscle diseases.

> While scientists have achieved success in targeting therapeutic genes

> to 

> nuclei, it's more challenging to get genes into mitochondria. The

> problem lies 

> in the gene delivery vehicles, or vectors, says Volkmar Weissig, a

> biochemist

> at  Northeastern University in Boston.

> In gene therapy, a vector's first task is to penetrate the cell's

> outer 

> membrane, or skin. Once inside the cell, the vector must breach a

> second  membrane

> surrounding either the nucleus or the mitochondrion, all the while 

> holding

> on to its genetic payload.

> The most commonly used vectors for gene therapy are viruses and

> synthetic 

> compounds called liposomes, hollow spheres composed of an outer shell

> of lipid 

> (the same type of chemical that makes up cell membranes). Viruses,

> says

> Weissig,  naturally infect cells and introduce foreign genes into

> nuclei, but

> apparently  can't send genes to mitochondria. With a composition

> similar to cell

> membranes,  liposomes can penetrate cells, but they usually release

> their DNA

> into the  cellular space shortly afterward.

> To make mitochondrial gene therapy feasible, Weissig is using MDA

> support to 

> custom-design a vector from a lipidlike, antiseptic chemical called

> dequalinium  (DQA). A single unit of DQA looks like a traditional

> Native American

> weapon  called a bola, and this bolaform shape allows DQA to form

> liposome-like

> capsules  called DQAsomes. DQAsomes can be filled with DNA, and thus

> might be

> ideal  weapons against mitochondrial diseases, Weissig suggests. So

> far, he's

> shown  that the DQAsomes can selectively target DNA to mitochondrial

> membranes.

> " This is basic research " that won't immediately lead to treatment for 

> mitochondrial disease, Weissig says. But it's a significant step in

> the right 

> direction.

>

>

>

[Guest guest]

Sorry, the reference does not seem to get printed right here.

When you get to PubMed, just type in the author's first name, leave one

space, and then initial. The paper is among the first.

> http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?

> cmd=Retrieve&db=pubmed&dopt=Abstract&list_uids=15384945