Penn Researchers ‘Edit’ Genes to Make Cells Resist HIV

Marie McCullough, Philly, March 6, 2014

University of Pennsylvania researchers have snipped out a single gene in patients’ immune cells to make them partly resistant to infection with HIV, the virus that causes AIDS.

The study, in this week’s New England Journal of Medicine, bolsters hope for controlling HIV without daily antiviral drugs–a so-called functional cure.

But even more important, as the first paper to report the modification of an exact spot in human DNA, it marks the arrival of the age of gene editing.

The researchers’ editing tool, developed by Sangamo BioSciences of Richmond, Calif., was made of natural proteins that recognize specific DNA sequences. These “zinc finger nucleases” can be used like molecular scissors to introduce intentional genetic mutations.

Until now, gene therapy has relied on disabled viruses to carry and dump genes somewhat randomly into a cells’ DNA.

“The ability to edit the human genome has been a prayer ever since we first understood that genes control biology,” said Sangamo CEO Edward Lanphier, who founded the company in 1995. “But we’ve moved beyond the concept of gene replacement, which was the idea behind gene therapy. Gene editing is much safer and more effective.”

Eminent AIDS researcher Anthony S. Fauci, director of the National Institute of Allergy and Infectious Diseases, said, “I think this is an important step in the right direction, not only for HIV, but for other diseases.”

Indeed, Sangamo is working on zinc finger-based approaches to treat and possibly cure hemophilia, Huntington’s disease, and sickle-cell anemia–diseases caused by a single defective gene. {snip}

{snip}

The Penn study–led by gene-therapy pioneer Carl June and underwritten with federal and Sangamo funding–built on the observation that some people are naturally invulnerable to HIV infection because of genetic variations. When they do get infected, their progression to AIDS is unusually slow.

One such gene variant keeps T cells–the disease-fighting blood cells that HIV attacks–from making a receptor, a sort of doorway, that HIV uses to break in. About 10 percent of Caucasians have inherited one copy of this gene variant, making them resistant to HIV infection. About 1 percent have two copies–one from each parent–making these fortunate few immune to HIV.

To artificially confer this invulnerability, June’s team removed T cells from 12 HIV-infected patients who were taking standard antiviral drugs and used zinc fingers to delete the doorway receptor gene. The modified T cells were coaxed to multiply, then each patient received a transfusion of roughly 10 billion.

Zinc finger editing is not perfect–at least, not yet–so only about 20 percent of the modified cells actually lacked the doorway receptor gene.

To see whether these modified cells might be fighting HIV, the researchers interrupted standard drug therapies for three months in six patients with healthy T cell counts. (The other six patients had suboptimal T cell counts despite standard drugs, so their treatment was not interrupted.)

Blood levels of HIV decreased in four of the patients who suspended treatment, falling to an undetectable level in one man.

A single patient had a serious complication, but it was a reaction to the transfusion, not the cells.

Some of the modified T cells were found to concentrate in the patients’ guts, where HIV builds a stockpile. Current drugs reduce the ability of HIV to reproduce but can’t completely eliminate it from the body because of this reservoir.

{snip}

Topics: , , ,

Share This

We welcome comments that add information or perspective, and we encourage polite debate. If you log in with a social media account, your comment should appear immediately. If you prefer to remain anonymous, you may comment as a guest, using a name and an e-mail address of convenience. Your comment will be moderated.
  • Pro_Whitey

    Whitey saves the world (and gets no credit).

    • Worse than no credit. Whitey is hated for his humane nature. You’ve seen the mau mau mutilations of whites in South Africa, so you know what I mean.

    • exlib93

      …took the words out of my mouth.

    • sbuffalonative

      YT will be paying for his kindness with this own extinction when he’s overwhelmed by the people he saved.

      • Gulp, that’s a good point!

      • 1stworlder

        If it wasn’t for food and medicine sent to Africa it wouldn’t be overpopulated.

  • “Race is just a social construct, with no biological validity.”

    Risk factors for osteoporosis.

    • 1stworlder

      Opportunity cost these guys could have cured a disease of whites instead. That said there are not a lot of blacks & Hispanics that live past 80 but they sure can vote past 110, so whites and Asians will have more of the diseases of old age. Part of obongo care was to rob from the old and give to the illegals.

    • I take Citracal.

  • IstvanIN

    I am ashamed to say but I am iffy on this. On the one hand anything that advances science and saves lives or prevents disease is wonderful. On the other hand they will give this treatment free to African, just like every other health advance we have made (and they never could) and their population will explode at an even faster rate.

    • 1stworlder

      It wont be free whitey will pay for it.

      • IstvanIN

        Free to them.

    • DaveMed

      Last year, we had a small group in which the leader told us with a smirk that developing countries were working out ways to obtain drugs for HIV and malaria that were not strictly legal (that is, using illegal knockoffs).

      I couldn’t say anything, but all I could think about was all the reckless sex and out-breeding of Whites that this would lead to. Not to mention the exponential rise in tax dollars we will have to pay.

      Quite an infuriating experience.

      • IstvanIN

        But they don’t take the drug properly which could lead to uncontrollable mutations.

  • Oil Can Harry

    I’m waiting to see if scientists in Africa can stop AIDS.

    Their plan is to isolate the virus in a laboratory and then throw a spear at it.

    • Spartacus

      Well after their groundbreaking scientific success of proving homosexuality is bad using magnets – the sky is the limit .

      • 1stworlder

        There is actually a picture of a black guy that proved gay marriage is possible with magnets.

    • BaronBaal

      Actually, you’re totally ignorant of the matter. Being proud of your race and heritage is one thing but being a total idiot is another. There are highly intelligent people of all races- including blacks, and many top scientists are African blacks. As for HIV and AIDS, there are plenty of powerful herbal medicines that black Africans have been using for thousands of years, and blacks scientists are making major breakthroughs in HIV/AIDS because of the vast availability of African herbal medicines. And do you know what? Every time they make a breakthrough big US pharma rushes there to see the research so that they can take the research results, get a patent, and monopolize whatever herbal medicine was involved, and take credit for the “discovery” and profit whenever or if they can make it into a new vaccine or possibly a cure. This has happened a lot in the past, and “Big US Pharma” literally FORCES its HIV drugs on black Africans while trying to discredit the usefullness of herbal African medicines, and only stops doing so when the evidence is so overwhelming that the herbal medicines do stop HIV/AIDS, and they then do what I stated above. Now, I understand your disdain with black Americans because these people live in close proximity to you and (can) threaten your way of life but black Africans have done NOTHING to you so grow up and mature up…

      • jambi19

        Name one. Better yet give me a source.

        • BaronBaal

          Done.

      • Oil Can Harry

        Let’s make a deal: If, God forbid, you or I ever become infected with HIV you can fly to Africa and ask the Bushmen to give you their wonder herbs which supposedly treat AIDS.

        As for me, I’ll pay “Big Pharma” for their drug cocktails, the same ones that have kept thousands of people with AIDS like Magic Johnson alive for decades.

        • BaronBaal

          The latest 2 herbs from Africa that have caught the eye of big pharma are Geranium root extract and Nigella sativa (aka black cumin seed). One HIV+ African took 10 mg of Nigella sativa daily and his viral load was undetectable after 6 months. Now here’s the eye-opener: The African took no anti-retroviral drugs or other herbal medicines for an entire year, and when he was retested after that year, no HIV could be found in his body. And this news is only a few days old so you can use any search engine and find out for yourself. Yes, anti-retrovirals do extend lives but there’s a catch to it. Most HIV+ people these days don’t die of AIDS at all. Rather, they die of heart attacks and liver failure because of the toxicity of the anti-retroviral drugs. Now, I’m not telling anyone to stop their HIV medications but I am saying that one should not overlook the potential of herbal remedies. If “Big Pharma” were the “masters of the universe”, so to speak, we would have had a cure by now, no? But 30 years on they are only just beginning to understand the mechanisms that allow HIV to harm the immune system in the manner it does.

          • jambi19

            Well one case and now it’s a proven fact! Do you understand what observer bias is? Confounding variables? Guess what???? HIV+ individuals die from just about everything in extremely high numbers because….they are immunodeficient. There is much more in the treatment of HIV than mere viral load. Such as opportunistic infection prevention, sterilization, screening, and etc. Let’s just call it modern western (white) medicine and stop giving credence to witch doctors that even if they are correct would never NEVER be able to produce and manufacture a cure like evil Big Pharma. These people can’t even eradicate malaria and polio from their defunct societies and now they will cure HIV. That notion is offensive to any honest intellectual. Research the Berlin Patient.

          • BaronBaal

            Jambi, you’re an idiot. Herbal medicine IS medicine- it is medicine in its NATURAL FORM. Do you think that your big pharma buddies just make medicine out of thin air? You know nothing about medicine or the pharmaceutical industry so you should just shut up. And your racism towards blacks is pathetic. I don’t particularly care for the company of blacks either but that doesn’t mean I hate African blacks just because they’re black or somewhat less “advanced” than I am. You’re pathetic.

  • So CAL Snowman

    Approximately 10% of the White population are immune to HIV. Their immunity is due to a mutation of the CCR5 gene. The CCR5 gene is a protein on the surface of White blood cells which the HIV virus uses to enter and infect host cells. A mutation of CCR5 known as CCR Delta 32, protects the white blood cells from HIV infection. It was speculated that the CCR Delta 32 mutation was favored by natural selection during the Black Death in Europe. However new studies show that is was actually small pox (or a variant thereof) that naturally selected for the CCR Delta 32 mutation in Europeans.

    • 1stworlder

      So if griods get this gene via therapy they could screw around until they get an HIV virus that can affect us.

      • jambi19

        correct there are certain mutated HIV strains in which CCR5 offers no protection.

        • BaronBaal

          Name one. Or better yet, give me a source.

          • jambi19

            Does tropism mean anything to you? Perhaps CCR5 would offer “resistance” but that is not the same this as “immunity” is it? You don’t think there would be selective pressures the virus would mutate? Are you confident it is resistant to superinfection? Or will your voodoo doctor spices and herbs cure that too?

    • BaronBaal

      Have a source?

  • JohnEngelman

    “The ability to edit the human genome has been a prayer ever since we first understood that genes control biology,” said Sangamo CEO Edward Lanphier.

    – Marie McCullough, Philly, March 6, 2014

    Genes also influence ability levels and behavior. It is still dangerous to point that out, however.

  • Spartacus

    I’d be happy if any potential cure were kept away from Africa…

    • newscomments70

      I recall reading an article about German researchers testing a vaccine for Type 2 AIDS, which is found in Africa. In the West, Type 1 AIDS is more common. They had zero interest in researching TYPE 1 AIDS. They only wanted to help Africans, not whites. This act epitomizes European liberalism.

  • dd121

    The AIDS virus was designed to attack the black genome more readily than that of whites. There is some spillover but that was deemed acceptable.

    • 1stworlder

      Yea and the air force fly’s crack cocaine into cities forcing blacks to smoke it. C130s landing on MLK boulevard dropping off free crack.

      • IstvanIN

        That is just foolish, they bring it in in vans.

        • PvtCharlieSlate

          Pimpmobiles.

      • dd121

        I know it’s hard to believe. It’s not important that you do.

    • Bob Wallace

      “Preposterous” means “having your head up your butt.” You might want to keep that in mind.

    • The virus was native to Africa, so of course it attacks Africans and other hominid species there more easily. They also aren’t acclimated yet. Plague – Yersina pestis – originated near Lake Balkash. The Plague of Justinian in 512 AD was bubonic plague. The Plague of Athens probable was not. Eurasians are now only a little bit acclimated to plague. The problem for higher animals is that we reproduce more slowly, so it takes more time for us to develop pan-resistance. Microbes reproduce rapidly, so mutations that offer them any sort of competitive advantage when it comes to infecting us become dominant in a very quick – to us – timespan.

      Ultimately, higher animals become resistant to a particular disease, but the disease also stops killing its host. We co-evolve. What we are seeing with HIV is ugly, but not unusual in biology.

      • dd121

        Yeah, I know. I have porphyria which is a genetic blood disorder. It conveys resistance to bubonic plague. With the waves of plague that has Europe over the centuries the gene has increased in frequency. Now penicillin will cure it.

  • DaveMed

    I believe you can. Haven’t read the study, but they’re probably referring to the CCR5 deletion, for which tests are available.

  • IstvanIN

    No women, no Asians, no Latinos and NO BLACKS? the research was either faked or stolen.

    • newscomments70

      Don’t worry, the movie version will feature a diverse team. The “corrected” team will be all non-white except for one nerdy white male who screws everything up; Morgan Freeman will save the day.

      • Or Samuel L Jackson,
        “I’m tired of these MFing Aids in these MFing patients!”

    • saxonsun

      So you really believe that all the female scientists succeeded because of AA? I suppose it comforts you terrified tiny males to believe that women are less intelligent than men. Funny!

      • IstvanIN

        Why yes I do, take Marie Curie for instance, Affirmative Action all the way

      • Of course it is funny! I got 79 US and foreign patents awarded in microelectronics production chemical methods in six years, ma’am. The stress level involved also got me a blood pressure of 175/105 by the time I was 31. Now at the age 48 finish wire, it is 85/45. If you want to go do what I used to do, that’s fine, but I’m staying right here at home.

        He who laughs last, laughs best.

  • DieWulfe

    Well, it’s nice to know Lanphier is French, June English and Fauci Italian. One less you know who to win a Nobel Prize.

  • William Allingham

    we can attribute one invention to black people and that is AIDS (involuntarily though) it seems that in Africa they from time to time have sex with apes, apes have developed immunity to the virus since they have had it since a long time but it mutated into human form and blacks spread it across the world, how could someone have sex with apes is perplexing but maybe they didn’t knew they were dealing with another species (multiculturalism on its finest expression).

  • Zaporizhian Sich

    If genes can be “edited” to thwart HIV, you can bet synthetic disease organisms can be created to kill only people of specific racial or ethnic groups. The deployment of this sort of bioweapons is a real possibility against us, not that darkies aren’t bioweapons already.

    • Ken Alibek wrote about chimerical disease weapons after he defected to the United States. His book “Biohazard” was sickening. ISBN 0-375-50231-9.

  • Xian Saon

    Greetings to everyone here, I’m here to say thanks to Okoh the man who helped me to cure my HIV disease. I was infected by someone i loved and he did not tell me he was HIV positive. i was searching through the internet for help and i saw a comment of people speaking on how Okoh has help to cure their HIV disease and i contacted Okoh through his email [email protected]m and i was told what to do. To my greatest surprise after patiently going according to instruction given to me by Okoh, i went for a medical test and the result was negative. I want to say thank you Okoh and God will bless you with long life for the good work you are doing. you can contact Okoh also via his e-mail: [email protected] or telephone: +2348153089532

  • Benatia Lopez

    Contact Dr. Oba today if you need cure to HIV ,
    Don’t bother yourself anymore, you have finally found the cure, Contact
    Dr. Oba on [email protected], or call him on +2347050254817, He will help
    you, he cured me from HIV, remember #HEALTH IS #WEALTH, Nothing
    good comes so easily, he will help you okay!!

    Contact Dr. Oba today if you need cure to HIV ,
    Don’t bother yourself anymore, you have finally found the cure, Contact
    Dr. Oba on [email protected], or call him on +2347050254817, He will help
    you, he cured me from HIV, remember #HEALTH IS #WEALTH, Nothing
    good comes so easily, he will help you okay!!

    Contact Dr. Oba today if you need cure to HIV ,
    Don’t bother yourself anymore, you have finally found the cure, Contact
    Dr. Oba on [email protected], or call him on +2347050254817, He will help
    you, he cured me from HIV, remember #HEALTH IS #WEALTH, Nothing
    good comes so easily, he will help you okay!!

  • Amanda Philip

    Hello!! I’m indeed very happy for the great help that Dr. Oraede rendered to me, I was a HIV patient my husband also was a HIV patient, we saw a blog whereby Dr. Oraede cured HIV, we (Me and My Husband) decided to contact him which we did, he asked us to buy some items, unfailingly we sent him the money he will need in buying the items required, He casted the spell and asked us to go for check-up after three days of casting the spell, Luckily for us we were tested HIV negative, now I believe all these Testimonies about him on the internet, he is truly a great man, if you want to discuss with me on how he cured us, kindly email me on [email protected] or you can contact the great Herbalist and a spell caster on [email protected] [email protected] [email protected] or you can call him via his mobile phone number on +2349038604218..

  • Amanda Philip

    Hello!! I’m indeed very happy for the great help that Dr. Oraede rendered to me, I was a HIV patient my husband also was a HIV patient, we saw a blog whereby Dr. Oraede cured HIV, we (Me and My Husband) decided to contact him which we did, he asked us to buy some items, unfailingly we sent him the money he will need in buying the items required, He casted the spell and asked us to go for check-up after three days of casting the spell, Luckily for us we were tested HIV negative, now I believe all these Testimonies about him on the internet, he is truly a great man, if you want to discuss with me on how he cured us, kindly email me on [email protected] or you can contact the great Herbalist and a spell caster on [email protected] [email protected] [email protected] or you can call him via his mobile phone number on +2349038604218