Gene editing genes in human hematopoietic cells by CRISPR/Cas9 system
Genome editing (also called gene editing) is a group of technologies that give scientists the ability to change an organism's DNA. These technologies allow genetic material to be added, removed, or altered at particular locations in the genome. Several approaches to genome editing have been developed. A well-known one is called CRISPR-Cas9, which is short for clustered regularly interspaced short palindromic repeats and CRISPR-associated protein 9. The CRISPR-Cas9 system has generated a lot of excitement in the scientific community because it is faster, cheaper, more accurate, and more efficient than other genome editing methods.
In 2015, scientists successfully used somatic gene therapy when a one-year old in the United Kingdom named Layla received a gene editing treatment to help her fight leukemia, a type of cancer. Doctors tried many treatments before this, but none of them seemed to work, so scientists received special permission to treat Layla using gene therapy. This therapy saved Layla's life. However, treatments like the one that Layla received are still experimental because the scientific community and policymakers still have to address technical barriers and ethical concerns surrounding genome editing.
Programmed death protein 1 (PD1) is a common immunosuppressive member on the surface of T cells and plays an imperative part in downregulating the immune system and advancing self-tolerance. Its ligand programmed cell death ligand 1 (PDL1) is overexpressed on the surface of malignant tumor cells, where it binds to PD1, inhibits the proliferation of PD1-positive cells, and participates in the immune evasion of tumors leading to treatment failure. The PD1/PDL1-based pathway is of great value in immunotherapy of cancer and has become an important immune checkpoint in recent years.
We already declared two patents on PD1 KO and target CD19 or MSLN CAR-T development.These gene editing targets are:
In 2015, scientists successfully used somatic gene therapy when a one-year old in the United Kingdom named Layla received a gene editing treatment to help her fight leukemia, a type of cancer. Doctors tried many treatments before this, but none of them seemed to work, so scientists received special permission to treat Layla using gene therapy. This therapy saved Layla's life. However, treatments like the one that Layla received are still experimental because the scientific community and policymakers still have to address technical barriers and ethical concerns surrounding genome editing.
Programmed death protein 1 (PD1) is a common immunosuppressive member on the surface of T cells and plays an imperative part in downregulating the immune system and advancing self-tolerance. Its ligand programmed cell death ligand 1 (PDL1) is overexpressed on the surface of malignant tumor cells, where it binds to PD1, inhibits the proliferation of PD1-positive cells, and participates in the immune evasion of tumors leading to treatment failure. The PD1/PDL1-based pathway is of great value in immunotherapy of cancer and has become an important immune checkpoint in recent years.
We already declared two patents on PD1 KO and target CD19 or MSLN CAR-T development.These gene editing targets are:
- Programmed cell death protein 1(PD1)
- T cell receptor alpha (TCR-α)
- CD52
Each of the three targets has completed the animal test study in China.
The combination of KAEDI’s expertise in immunotherapy and antibody development with editing experience in CART development and commercial cGMP manufacturing makes KAEDI as a major participant in the biopharmaceutical industry to increase access to effective Gene Editing.
The combination of KAEDI’s expertise in immunotherapy and antibody development with editing experience in CART development and commercial cGMP manufacturing makes KAEDI as a major participant in the biopharmaceutical industry to increase access to effective Gene Editing.