Gene Therapy (Zinc-finger nuclease, Crisper-CAS 9)

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Presentation transcript:

Gene Therapy (Zinc-finger nuclease, Crisper-CAS 9)

Genetic Basis of Disease SICKLE CELL ANEMIA IS CAUSED BY A SINGLE POINT MUTATION ON A SINGLE GENE.

CORRECT MUTATION = CURE DISEASE!

Zinc Nucleases

Gene Therapy Attempt to correct diseases caused by a single defective gene by transplanting cells with healthy copies of gene = mutated gene = normal gene Intro video 1st 2 min good

Gene Therapy Corrected version of gene is inserted in VECTOR – most often vector is a modified VIRUS such as Adenovirus or Lentivirus. New generation viruses are unable to replicate, and are engineered to reduce immune system response. Adenovirus can be expressed by injection into nucleus without being incorporated into chromosome (avoids incorrect insertion into normal genes).

Challenges of Gene Therapy- What could possibly go wrong? Theory is wonderful – cure disease by correcting genetic mistakes or introducing new beneficial protein – practically very difficult to do Gene Therapy is very high risk Only used for life-threating diseases known to be caused by a single gene

Challenges of Gene Therapy- What could possibly go wrong? (1999) IMMUNE SYSTEM REJECTION – 1 case of 19 year old male dying 4 days after start of therapy due to massive immune system response (Early 2000’s) Random insertion of viral DNA into healthy genes – 3 cases of patients developing leukemia Will transformed cells reproduce and successfully compete with abnormal cells? If not therapeutic impact is only temporary. Regulation of gene expression – how can we get inserted DNA to be expressed in appropriate amounts at the appropriate times?

Severe Combined Immunodeficiency (SCID) Immune cells do not develop “Bubble Boy” – children with SCID have been isolated in “bubbles” or mini space suits to avoid exposure to disease

SCID animation Dana Farber video Procedure used for treating patients with SCID – immune deficiency disease. This disease is caused by MUTATION IN ONE GENE AND IS USUALLY FATAL. 6 PATIENTS “CURED”.

Cure for Blindness: Gene Therapy reverses sight loss and is long-lasting 2016: Significant sustained improvement over two years in vision for 32 patients in UK, US, Canada and Germany

Hemophilia (Blood clotting disorder) 2017 Video CNBC Nov. 2018 April 2018- First Successful Gene Therapy for Hemophilia A Induces Normal or Near Normal Blood clotting Factor VIII Levels for 11 out 13 patients

Immunotherapy – activating immune system to fight cancer is currently biggest area of gene therapy research 2018 Nobel Prize in Physiology and Medicine awarded to James Allison and Tasuku Honjo for developing field of immunotherapy – exploring ways to activate immune system against cancer.

HALLMARKS OF CANCER CELLS

IMMUNOTHERAPY: CAR T targets CD-19 protein common on surface of Non-Hodgkin’s Lymphoma and acute lymphoblastic Leukemia cancers CAR T immunotherapy

Frontiers of Gene Therapy: Using CRISPR-cas9 to fix disease in cells Stage 1 Hunter’s in vivo 2018 PRACTICAL CONCERNS: OFF-SITE CUT PROBLEM (CUTS OR INSERTS IN WRONG PLACE) HAS NOT YET BEEN SOLVED ETHICAL CONCERNS: SHOULD WE PURSUE TECHNOLOGY THAT COULD LEAD TO “DESIGNER” BABIES – SELECT SPECIFIC TRAITS

Hunter direct injection