Gene Therapy - GEC 1 (Reviewer)

History of Gene Therapy

• it was first conceptualized in 1972

• first successful gene transfer was shown on a trial in 1990 to treat adenosine deaminase deficiency-severe combined immunodeficiency (ADA-SCID).

• The first somatic gene therapy was documented for malignant brain tumors by using recombinant DNA technology which makes the tumor cells sensitive to drugs and causes the tumor cells to die

• In 1990, the first approved gene therapy clinical trial took place when Ashanthi De Silva, a 4 year old girl with ADA-deficiency, was given her own T cells engineered with a retrovial vector carrying a normal ADA gene.

Vector

to insert new genes directly into cells, scientists use a vehicle called “vector” which is genetically engineered to deliver the gene

Virus as Vector

• viruses have a natural ability to deliver genetic material into cells

• it is modified to remove its ability to cause an infectious disease

Gene Therapy

an approach of treating diseases by either modifying the expressions of an individual’s genes or correction of abnormal genes

Types of Gene Therapy

1. Somatic Gene Therapy

2. Germ Line Therapy

Somatic Gene Therapy

targets specific cells and tissues, without affecting the germline, ensuring that the changes are not passed down to future generations

Germ Line Gene Therapy

alters the genes of reproductive cells, potentially impacting future generations. This approach is currently ethically debated and not widely used

Gene Editing

type of genetic engineering in which DNA is inserted, deleted, or modified in the genome of a living organism

How gene therapy works?

1. They can replace a gene that is missing or is causing a problem.

2. They can add genes to the body to help treat disease.

3. They can turn off genes that are causing problems.

Two Types of Gene Therapy

1. In VIVO Gene Therapy

2. Ex VIVO Gene Therapy

In VIVO Gene Therapy

when gene therapy is used to modify cells inside the body, a doctor will inject the vector carrying the gene directly into the patient.

Ex VIVO Gene Therapy

when gene therapy is used to modify cells outside the body, doctors take blood, bone marrow, or another tissue, and separate specific cell types in the lab.

Gene Therapy Delivery Methods

1. Viral Vectors

2. Non-Viral Vectors

3. Direct Gene Transfer

Benefits of Gene Therapy

• Potential for a cure

• One-time treatment

• Treating incurable disease

Challenges in Gene Therapy

• Vector and safety immune response

• Inefficient or off-target delivery

• Durability and expression

Stem Cell

• building blocks of the human body

• they are needed for the maintenance of tissues as well as for repair after injury

Applications of Gene Therapy

• Cancer

• Inherited Diseases

• Infectious Diseases

Ethical Considerations

• Safety

• Equity and Access

• Enhancement vs. Treatment

Future Directions

• Improved Delivery Methods

• Expanding Applications

• Personalized Medicine

Impact on Society

• Improved Health Outcomes

• Enhanced Research

• Global Impact