HIV/AIDS Breakthrough Via Gene Therapy
Right now, more than 35 million persons worldwide have the HIV virus.
For the last few decades, anti-retro viral drugs have kept many of them alive, living with the virus rather than dying of AIDS.
For more than 1.
5 million who died this year though, AIDS still won out.
It was announced by researchers in the New England Journal of Medicine that the results of a phase I study regarding the use of genetic editing shows great promise for HIV patients.
This is the first successful example of genetic editing in humans.
The gene therapy is used not to destroy the HIV virus as current medications do, but is used instead to assist the patients' own immune systems to fight HIV.
This phase involved 12 patients who were diagnosed with HIV.
In this study, patients' white blood cells were removed, their genes edited, then 10 billion modified cells were infused back into each patient.
After reintroduction of the modified cells, six patients showed such a small viral load that they were taken off all HIV drugs altogether.
One patient, who subsequently showed no HIV at all, was later found to have a natural CCR5 mutation.
The study was done in partnership in partnership with the University of Pennsylvania (Penn), P.
A.
, Albert Einstein College of Medicine, Bronx, N.
Y.
, and Sangamo BioSciences, Richmond, C.
A.
Sangamo developed the gene editing technology, called zinc-finger nuclease (ZFN), described by researchers as virtual molecular "scissors.
" The ZFN technology was used to edit the gene called CCR5 in the immune T cells to mimic a mutation that occurs naturally in one percent of humans.
This mutation, CCR5-delta 32, enables the affected population to exhibit a natural resistance to HIV.
While current HIV medication was developed to destroy the virus, this mutation denies the virus a way to enter the immune cells in the first place.
The purpose of this first phase was to gauge the safety of the treatment and patients' tolerance.
This first phase is considered a success.
The infusions were seen to be safe and tolerable.
The modified T cells continue to remain in patients during follow-up checks.
Next, more patients will be tested using a larger number of edited T cells to find better ways to assist the modified cells to persist in a patient's body to achieve as near a cure as possible.
Scientists hope to use this genetic therapy to achieve a "functional cure"
For the last few decades, anti-retro viral drugs have kept many of them alive, living with the virus rather than dying of AIDS.
For more than 1.
5 million who died this year though, AIDS still won out.
It was announced by researchers in the New England Journal of Medicine that the results of a phase I study regarding the use of genetic editing shows great promise for HIV patients.
This is the first successful example of genetic editing in humans.
The gene therapy is used not to destroy the HIV virus as current medications do, but is used instead to assist the patients' own immune systems to fight HIV.
This phase involved 12 patients who were diagnosed with HIV.
In this study, patients' white blood cells were removed, their genes edited, then 10 billion modified cells were infused back into each patient.
After reintroduction of the modified cells, six patients showed such a small viral load that they were taken off all HIV drugs altogether.
One patient, who subsequently showed no HIV at all, was later found to have a natural CCR5 mutation.
The study was done in partnership in partnership with the University of Pennsylvania (Penn), P.
A.
, Albert Einstein College of Medicine, Bronx, N.
Y.
, and Sangamo BioSciences, Richmond, C.
A.
Sangamo developed the gene editing technology, called zinc-finger nuclease (ZFN), described by researchers as virtual molecular "scissors.
" The ZFN technology was used to edit the gene called CCR5 in the immune T cells to mimic a mutation that occurs naturally in one percent of humans.
This mutation, CCR5-delta 32, enables the affected population to exhibit a natural resistance to HIV.
While current HIV medication was developed to destroy the virus, this mutation denies the virus a way to enter the immune cells in the first place.
The purpose of this first phase was to gauge the safety of the treatment and patients' tolerance.
This first phase is considered a success.
The infusions were seen to be safe and tolerable.
The modified T cells continue to remain in patients during follow-up checks.
Next, more patients will be tested using a larger number of edited T cells to find better ways to assist the modified cells to persist in a patient's body to achieve as near a cure as possible.
Scientists hope to use this genetic therapy to achieve a "functional cure"