GENE EDITING

POTENTIAL CURE
OF AIDS
What is genome editing?

Genome editing technologies enable scientists to make changes to DNA, leading to


changes in physical traits, like eye color, and disease risk. Scientists use different
technologies to do this. These technologies act like scissors, cutting the DNA at a specific
spot. Then scientists can remove, add, or replace the DNA where it was cut.
The first genome editing technologies were developed in the late 1900s. More recently, a

new genome editing tool called CRISPR, invented in 2009, has made it easier than ever to
edit DNA. CRISPR is simpler, faster, cheaper, and more accurate than older genome
editing methods. Many scientists who perform genome editing now use CRISPR.
 What is genome editing used for?

• Genome editing could be used to edit the genome of any organism.

• It is against the law to use genome editing in human embryos that will be allowed to develop
beyond 14 days.
• Genome editing can be used:
• For research: Genome editing can be used to change the DNA in cells or organisms to understand their
biology and how they work.
• To treat disease: Genome editing has been used to modify human blood cells that are then put back into
the body to treat conditions including leukemia and AIDS. It could also potentially be used to treat other
infections (such as MRSA) and simple genetic conditions (such as muscular dystrophy and hemophilia).
• For biotechnology: Genome editing has been used in agriculture to genetically modify crops to improve
their yields and resistance to disease and drought, as well as to genetically modify cattle that don’t have
horns.
 CRISPR-Cas9:

 CRISPR-Cas9 was adapted from a naturally occurring genome

editing system that bacteria use as an immune defense. When
infected with viruses, bacteria capture small pieces of the
viruses' DNA and insert them into their own DNA in a
particular pattern to create segments known as CRISPR arrays.
The CRISPR arrays allow the bacteria to "remember" the
viruses (or closely related ones). If the viruses attack again, the
bacteria produce RNA segments from the CRISPR arrays that
recognize and attach to specific regions of the viruses' DNA.
The bacteria then use Cas9 or a similar enzyme to cut the
DNA apart, which disables the virus.
 Overview of HIV

 HIV (human immunodeficiency virus) is a virus that

attacks cells that help the body fight infection, making
a person more vulnerable to other infections and
diseases. It is spread by contact with certain bodily
fluids of a person with HIV, most commonly during
unprotected sex (sex without a condom or HIV
medicine to prevent or treat HIV), or through sharing
injection drug equipment.
 If left untreated, HIV can lead to the disease AIDS
What is AIDS?

 AIDS (acquired immunodeficiency syndrome) is the late stage of HIV infection that occurs

when the body’s immune system is badly damaged because of the virus.
 HIV infection with either a CD4+ T cell count below 200 cells per µL or the occurrence of
specific diseases associated with HIV infection is classified as AIDS
 HIV can transmit when body fluids containing the virus come into contact with a permeable
barrier in the body or small breaks in moist tissues of areas, such as the genitals.
 Specifically, HIV can transmit via:
• Blood, Semen ,Pre-seminal fluid, vaginal fluids, rectal fluid, breast milk
 A Bold Effort to Cure HIV—Using Crispr:

 An experiment tests whether the gene-editing technology can stop the virus from
replicating, which would ultimately wipe out the infection.
 HIV attacks immune cells in the body called CD4 cells and hijacks their machinery to make
copies of itself. But some HIV-infected cells can go dormant—sometimes for years—and
not actively produce new virus copies. These so-called reservoirs are a major barrier to
curing HIV.
 While antiretroviral drugs can halt viral replication and clear the virus from the blood, they
can’t reach these reservoirs, so people have to take medication every day for the rest of
their lives. But Excision BioTherapeutics is hoping that Crispr will remove HIV for good
 A Bold Effort to Cure HIV—Using Crispr:

 Crispr is being used in several other studies to treat a

handful of conditions that arise from genetic
mutations. In those cases, scientists are using Crispr to
edit peoples’ own cells. But for the HIV trial, Excision
researchers are turning the gene-editing tool against
the virus. The Crispr infusion contains gene-editing
molecules that target two regions in the HIV genome
important for viral replication. The virus can only
reproduce if it’s fully intact, so Crispr disrupts that
process by cutting out chunks of the genome.
Ethical obligations with genome editing:
Ethical concerns arise when genome editing, using technologies such as CRISPR-
Cas9, is used to alter human genomes. Most of the changes introduced with
genome editing are limited to somatic cells, which are cells other than egg and
sperm cells (germline cells). These changes are isolated to certain tissues and are
not passed from generation to generation. However, changes made to genes in egg
or sperm cells or an embryo’s genes could be passed to future generations.
Germline cell and embryo genome editing bring up several ethical challenges,
including whether it would be permissible to use this technology to enhance
normal human traits (such as height or intelligence). Based on concerns about
ethics and safety, germline cell and embryo genome editing are currently illegal in
the United States and many other countries.