Gene Editing 101
A SINGLE stranded chain that is essentially transcribed from DNA and translates into proteins!
RNA
A technique where a faulty gene is recognized and a carrier molecule (or vector) with the correct form of DNA will override the faulty gene and replace it with the correct copy when introduced to the body.
Gene Replacement Therapy
This is genetically engineered vehicle used to carry and deliver therapeutic genetic material into a cell
Name the ways where gene therapy can be delivered?
IV, Injection directly to the tissue/organ, Injection into a sample of the tissue/organ, and then returned to the patient.
Deoxyribonucleic acid- a double stranded helix present in all of our cells that carries our genetic information
DNA
What type of disease is ideal for gene replacement therapy?
Monogenic diseases! There is a mutation in a single gene that causes the features of a disorder or syndrome.
These are commonly used as vectors because they can deliver the material by infecting a cell
Viral Vectors
This method of gene delivery allows for the treatment to enter the patients' bloodstream.
IV Gene Therapy
Genepordy Activity! Listen closely, comprehension question at the end!
N/A
What type of genetic change is gene replacement therapy most useful?
Loss of function mutations since viral vectors can then replace the missing protein products.
List three types of vectors that can be utilized to deliver genes into the cell.
Adenovirus, retrovirus, lentiviral vectors, poxviruses, non-viral vectors based on plasmid (includes naked-DNA and liposomes)
This type of gene therapy involves the treatment of cells that do not produce sperm or eggs:
somatic gene therapy
Technology that allows us to modify DNA including adding, deleting, or altering the DNA
Gene Editing
What is an advantage of gene replacement therapy?
Provides options for patients with no other options.
Typically only given in one dose.
Long lasting effects.
This viral vector has a low pathogenicity, is able to deliver genetic material to highly diverse range of cell types, and is highly predictable with a reliable insertion pattern.
Adeno-Associated Virus (AAV)
This type of gene therapy involves the treatment of cells that DO produce sperm or eggs
germline gene therapy
A fast, accurate and cheap way to edit the genome in hopes of preventing or treating disease using a guide RNA that attaches to a target sequence in the genome.
CRISPR/CAS9
What are the disadvantages of gene replacement therapy?
Expensive - The gene therapy for Spinal Muscular Atrophy is $2.1 million.
Gene therapy is relatively new and there are no guarantees.
Potentially dangerous if non-targeted cells are affected.
Ethical Issues
The use of this type of vector is commonly used as they are cheaper, easier to produce in large amounts, and have low immunogenicity.
Nonviral Vectors
Genepordy Activity! Listen closely, comprehension question at the end!
N/A