CRISPR

It was discovered from bacteria! It’s a segment of DNA containing short repetitions of base sequences, involved in the defence mechanisms of prokaryotic organisms to viruses.

Who is He Jiankui and why is he now famous?

He Jiankui is a 35 year old biophysics researcher and was born in China .

He is famous for creating the first genetically edited baby, he altered the genes of a twin to prevent getting HIV in the future.

Who is Jennifer Doudna and what is she most famous for?

Jennifer Doudna was born on February 19, 1964 in washington D.C. United States of America. She is the wife of Jamie Cate and attended Harvard Medical School (1989) Pomona (1985). She is known for First X-ray based structure of catalytic, CRISPR. Jennifer is Famous for her discovery alongside with Emmanuelle Charpentier who is a French microbiologist . She discovered a molecular tool called the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)

What genetic diseases can CRISPR already help humans treat?

List of genetic diseases that CRISPR can possibly treat:

1.Cancer- Specifically the cancer of the esophagus (Esophageal cancer) which is treated by modified T-cell therapy in China.CRISPR is used to modify the cells to remove the gene that encodes for a certain receptor,

2.Blood disorders- The first CRISPR trial in Europe with the purpose of treating beta-thalassemia, a blood disorder that affects the oxygen transport in the blood. CRISPR is used produce fetal hemoglobin, a natural form of the oxygen-carrying protein that binds oxygen much better than the adult form.

3.Blindness- Hereditary forms like blindness. The specific mutations causing the disease are known making it easy to instruct CRISPR-Cas9 to target and modify that gene.

4. AIDS- using CRISPR to cut the HIV virus out of the DNA of immune cells. This approach could bring the key advantage of being able to attack the latent form of the virus, which is inserted into our DNA and inactive, making it impossible for most therapies to target it.

5. Cystic fibrosis-a genetic disease that causes severe respiratory problems. CRISPR technology could help us get to the origin of the problem by editing the mutations that cause cystic fibrosis, which are located in a gene called CFTR.

6. Muscular dystrophy-Duchenne’s muscular dystrophy is caused by mutations in the DMD gene, which encodes for a protein necessary for the contraction of muscles. Children born with this disease suffer progressive muscle dystrophy in children.

7.Huntington’s-a neurodegenerative condition with a strong genetic component. The disease is caused by an abnormal repetition of a certain DNA sequence within the huntingtin gene. The higher the number of copies, the earlier the disease will manifest itself.

Using the bottom half of the image above describe in words two ways that the gene-editing tool CRISPR can change DNA strands.

way one: it can delete a gene. a gene is made up of DNA. it is inherited from parents. it’s role is to tell or instruct the organism to build a characteristic, which is in it’s phenotype. if you delete a gene or disrupt the instructions in the gene it would change the characteristic or phenotype, and maybe remove it?

way two: insert a gene. if you insert a gene you would be adding a new sequence and new instructions. therefore you would get a new phenotype that was different from before.

Is it true the gene-editing tool CRISPR has been used to:

  • Create hornless dairy cows? Yes - researchers at Recombinetics in Minnesota inserted a gene for hornlessness into a cow genome, which they cloned and then inserted into a cow. 5 cows were born, 2 which are still alive and are named Spotigy and Buri, they both show no signs of horn buds.
  • Return hearing to deaf mice? No - but they have found a way to prevent deafness in mice.
  • Remove HIV from living things? Yes - scientists led by Kamel Khalili, director of the Comprehensive NeuroAIDS Center at Temple University, successfully eliminated HIV gene from a mice and rats genome who were previously infected.
  • Edit out Huntington's disease from humans? No - but nearly, as they have edited out huntington’s disease from a cell
Jeff, Elisha, Dani, Sia, Lei CRISPR - Google Slides