2. Gene editing in human embryos
• Developmental biologist Fredrik Lanner first
researcher to modify the DNA of healthy
human embryos.
• to see how they regulate early embryonic
development
(Jelor Gallego, 2016- Karolinska Institute in Stockholm)
3. Gene editing in human embryos
contd…
specifically target genes crucial to normal embryonic growth
• The tool utilizes two molecules that focus on your individual
genes to make ultra-precise changes to your DNA.
Infertility
How to treat debilitating
conditions
Stem cells
Miscarriages
4. improve IVF success rates and reduce
miscarriages
Genetically modified children –
Designer babies
Scope
Risks
Helps in treating person with diabetics,
Parkinson & blindness
5. Gene editing in malarial mosquitoes
• The Bill and Melinda Gates Foundation- create a mosquito-killing
technology -relies on CRISPR gene editing
• The Gates-funded project, called Target Malaria
• Technique- gene drive- is a way to spread traits through wild
populations of animals- selfish genes
• total investment to $75 million
(Antonio Regalado , 2016-London)
6. • DNA of malaria mosquitoes altered - that would cause them to
become sterile within about 11 generations—or in about one year
• The plan is to disperse Anopheles gambiae mosquitoes harboring
selfish genes
• The gene drive could spread across a huge swath of territory, causing
mosquitoes to disappear and blocking transmission of the parasite
that causes malaria.
7. Risk
Released in the wild- a
gene drive could push
other species to
extinction.
Other scope
kill off invasive
species, including rats,
toads, or fish that take
over ecosystems and
can drive local species
to extinction
8. Other risks…
• Would removing mosquitoes upset
ecosystems
• Are we risking a genetic epidemic if the selfish
DNA should jump the species barrier to affect
other insects?
9. Gene-editing method shrinks cancer
• Shenzhen University, China-Dr Weiren Huang, 2016
• Crispr-Cas9 replaces harmful DNA with new code that kills
cancerous cells while leaving healthy ones unharmed
• Mice with the reprogrammed code developed tumours that
were much smaller than cancers in mice that did not get this
treatment
• Unclear yet whether the technique would work in humans.
10. Scope in Lung cancer treatment
• T cells – immune cells in blood
• T cell subset Treg cells
T 17 Helper cells
• Treg cells produce a protein PD- 1 which limits
cell capacity to launch an immune system
Healthy Human T Cell
West China Hospital of
Sichuan University
Treg cells
11. • Doctors will extract T cells, and then knock out the gene that
encodes the PD-1 protein using CRISPER cas9
The edited cells will be multiplied in the lab before being
reintroduced to the patients
Inject the modified cells back into the patient
12. Curing AIDS from CRISPR
• UMass Medical School – to make the HIV completely
harmless using CRISPR
• a very precise pair of scissors to go in and clip out all, or part
of, the HIV genome and reattach the severed ends of the
human genome
Wolfe, 2015
13. • they show that the system can protect cells
from reinfection and that the technology is
safe for the cells, with no toxic effects.
• This would be a step on the road to getting a
functional cure for HIV.”
Editor's Notes
Whether creating GM crops or making animals disease-resistant, gene-editing has always faced harsh criticism
One fear is how altering human DNA in embryos could recklessly create a fatal genetic disease that would exist for generations
Despite this, one researcher is attempting the most controversial type of gene editing on earth. Developmental biologist Fredrik Lanner is the first researcher to ever modify the DNA of healthy human embryos.
He is from stokhom.....and doing reasearch in editing genes in human embryos to see how they regulate early embryonic development. He’s specifically targeting genes crucial to normal embryonic growth to learn more about infertility, miscarriages, stem cells, and how to treat debilitating conditions.
He is using the popular CRISPR-Cas9 editing method. The tool utilizes two molecules that focus on your individual genes to make ultra-precise changes to your DNA
Cripr case editng tool modify the target gene specifically which are cruicial for normal embryonic frowth to learn more about
CRISPR is short for “clustered regularly interspaced short palindromic repeats.”
to learn more about infertility, miscarriages, stem cells, and how to treat debilitating conditions.
will use CRISPR to edit out key genes from the embryo, to try to identify the genetic faults which lead many women to repeatedly miscarry. The embryos will be allowed to develop for just a few days.
If we can understand how these early cells are regulated in the actual embryo, this knowledge will help us in the future to treat patients with diabetes, or Parkinson, or different types of blindness and other diseases,
to create a mosquito-killing technology that relies on CRISPR gene editing.
the National Academies of Sciences, Engineering, and Medicine in Washington, D.C., said gene drives were not yet ready for environmental release, but laid out steps that should be taken to test them safely, recommendations the Gates Foundation says it will follow.
Scientists from china conducted an experiment on mice have used a gene-editing tool to stunt tumour growth in mice.
tool into a method of treating genetic diseases that for the most part have no treatments today- eye disease, cancer, sickle-cell anemia, and Duchenne muscular dystrophy, among others.Trial studies ar going in progress
cells might also attack normal tissue, Mr. Lu said, this first phase of the trial aims to determine whether the approach is safe.
Antiretroviral drugs are very good at cBut patients on antiretroviral therapy who stop taking the drugs suffer a rapid rebound in HIV replication."ontrolling HIV infection
here’s still a lot more work to be done in getting this technique ready for something more advanced than human cells in a petri dish - particularly when it comes to perfect accuracy for the 'cutting' process - but it’s an exciting first step