History
This decade marked the conceptual birth of gene therapy as scientists began connecting gene defects to human diseases.
What are the 1970s?
This term refers to a circular DNA molecule inserted to prepare therapeutic gene constructs in the lab.
What is a plasmid?
Gene therapy for diabetes primarily targets this type of diabetes that involves autoimmune destruction of beta cells.
What is Type 1 Diabetes?
One major advantage of gene therapy is that it targets this aspect of disease instead of just treating symptoms.
What is the root genetic cause?
ADHD is linked to short versions of these two types of dopamine-related genes.
What are dopamine transporter genes and dopamine receptor genes?
In 1972, these two scientists published a paper that formally proposed the concept of gene therapy for human diseases.
Who are Theodore Friedmann and Richard Roblin?
Gene therapy can be delivered in two main approaches: directly into the body or by modifying cells outside the body. Name both.
What are in vivo and ex vivo?
Name one type of cell (other than pancreatic beta cells) researchers aim to genetically modify to produce insulin.
What are liver cells, gut cells, muscle cells, or stem cells?
Some gene therapies offer this kind of benefit, meaning patients may only need one treatment.
What are long-term or permanent effects?
This mental health condition is associated with the short variant of the serotonin transporter gene and increased risk under stress.
What is depression?
This young girl with SCID was the first person to undergo gene therapy in a clinical trial in 1990.
Who is Ashanti DeSilva?
This type of delivery vehicle is used to carry the therapeutic gene into a patient’s cells.
What is a vector?
Scientists use these gene regulatory elements to ensure insulin is produced only when blood sugar is high.
What are glucose-responsive promoters?
Name one major risk or disadvantage associated with viral vectors in gene therapy.
What is immune response or insertional mutagenesis?
Gene therapy could modify this gene, also associated with BPD, which regulates serotonin reuptake.
What is the SERT gene?
Who's death in a gene therapy trial led to a major setback in the field due to this complication.
Jesse Gelsinger
In ex vivo gene therapy, patient cells are commonly harvested and then wht do scientists do to them?
What is inject them directly into the body?
One challenge in insulin-producing gene therapy is that this autoimmune issue may still persist.
What is the immune system attacking newly introduced insulin-producing cells? (Immune Rejection)
Gene therapy is often classified under this type of tailored treatment approach based on a person’s DNA.
What is precision medicine?
This neurodegenerative disorder is being targeted by gene therapy to deliver dopamine-producing enzymes or protective factors like GDNF.
What is Parkinson’s disease?
Approved in 2017, this gene therapy became the first FDA-approved treatment for a rare inherited form of blindness.
What is Luxturna?
A patient is monitored for safety and the gene produces the correct protein is called?
What is expression and monitoring?
This gene-editing tool is being explored to improve the safety and long-term success of insulin gene therapy.
What is CRISPR?
This is one reason why access to gene therapy remains limited for many people around the world.
What is high cost or limited availability?
In addiction disorders, gene therapy might alter genes in this neural reward system.
What is the dopamine or opioid receptor pathway?