CRISPR Therapies in Clinical Trials - Group 2

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CRISPR Therapies in Clinical Trials - Group 2 作者: Mind Map: CRISPR Therapies in Clinical Trials - Group 2

1. Delivery

1.1. Viral Vectors

1.1.1. Retrovirus

1.1.2. Adenovirus

1.1.3. Lentivirus

1.1.4. Adeno-associated virus (AAV) vectors

1.2. Non-Viral Vectors

1.2.1. Gold Nanoparticles

1.2.2. Lipid-Mediated Transfection

1.2.3. Cell-Penetrating Peptides

1.2.4. DNA Nanoclews

1.2.5. Liposomes

1.3. Direct Transport

1.3.1. Co-Microinjection

1.3.2. Lance Array Nanoinjection

1.3.3. Electroporation

1.3.4. Hydrodynamic Delivery

2. Development

2.1. Potential diseased state to treat

2.1.1. Cancer Cut Cut genes that limit cancer killing effect Cut interfering genes Screening Direct vivo Indirect vivo Vitro Clone the sgRNA of chioce

2.1.2. Hereditary blindness 1/2000 people worldwide IRD (Inherited Retinal Degeneration)

2.1.3. AIDS Situation We have drugs to decrease viral replication "Surgical knives" Zinc finger nucleases (ZFN) Transcription Activator-like Effector Nucleaases (TALENs) CRISPR

2.1.4. The future Other probable uses in research Sickle cell disease Cystic fibrosis Huntingdon's disease

3. Clinical Trials Using in vivo & ex vivo delivery of CRISPR

3.1. Ex Vivo Gene Editing

3.1.1. Ex vivo editing = T cells expanded ex vivo and then transfused back into patient 2016 First clinical trial using CRISPR for cancer therapy Sichuan University’s West China Hospital in Chengdu in 2016 Other successful clinical trials include gene editing for the following conditions Bladder Cancer B-Cell Lymphoma Multiple myeloma Esophageal cancer

3.2. In Vivo Gene Editing

3.2.1. CRISPR/Cas9 targeting HPV Less implemented in clinical trials

4. Editing Gene Method

4.1. plasmid-based CRISPR-Cas9 system encoding the Cas9 protein and sgRNA from the same vector

4.1.1. Cas9/sgRNA complex inside cells

4.2. mixture of the Cas9 mRNA and the sgRNA.

4.2.1. Cas9 mRNA will be translated the Cas9/sgRNA complex

4.3. Cas9/sgRNA complex

5. Nuclear localization

6. Active Clinical Trials

6.1. Companies

6.1.1. Original developers have developed their own companies Caribou Biosciences by Charpentier CRISPR Therapeutics by Doudn Editas by Feng Zhang

6.1.2. Leading Biotechnology Companies AstraZeneca, DuPont Novartis Thermo Fisher Scientific Signma Aldrich

7. Side effects

7.1. Genetic Drive

7.1.1. Genetic Damage Complex rearrangements Unexpected large deletions Off-target alterations

8. What is CRSPR?

8.1. If the assigned RNA is found on the bacterium DNA, the DNA will be trimmed off

8.2. "Clustered Regularly Interspaced Short Palindromic Repeats"

8.3. Bateria's Anti-viral system

8.3.1. similar to immunological memory, but at DNA scale

8.3.2. Bacteria copy and store DNA segments inserted by the bacteriophage into CRSPR The segments are transcripted to RNA and assigned to Cas9

9. Cas-9

9.1. Cas9 scouts around for the assigned RNA

9.2. CRSPR-Associated protein 9

10. CRSPR-Cas9 Gene Editing

10.1. Cas9 can find and cut whatever RNA segments it is assigned

10.1.1. CRSPR-cas9 is a high-precision DNA cutting/inserting tool

10.2. DNA repair

10.2.1. NHEJ "Non-Homologous End Joining" The body's DNA repair mechanism will automatically join the two ends of the DNA strand previously cut by Cas9

10.2.2. HDR "Homologous Directed Repair" Adding a DNA template that the DNA repair mechanism can build on to join the two ends of the DNA strand previously cut by Cas9