Gene Editing – SunAct Cancer Institute

Therapies

What is Gene Editing Therapy?

Gene Editing therapy is an ability to make highly specific changes in the DNA sequence of a living organism, essentially customizing its genetic makeup.

How does Gene Editing Therapy work?

Gene Editing is performed using enzymes, particularly nucleases that have been engineered to target a specific DNA sequence, where they introduce cuts into the DNA strands, enabling the removal of exiting DNA and the insertion of replacement of DNA. Key among gene–editing technologies is a molecular tool known as CRISPR–Cas9.

Identification: Key genetic mutations or abnormalities associated with cancer are identified.
Editing: Advanced techniques, such as CRISPR-Cas9, are used to make precise changes to the patient’s DNA, correcting harmful mutations or enhancing specific genes.
Validation: Edited cells are tested to ensure that the changes are effective and safe.
Infusion: The corrected cells are reintroduced into the patient’s body, or gene editing is used to directly modify cancer cells to improve their response to treatment.

Benefits

Precision: Targets specific genetic mutations associated with cancer, potentially leading to more effective treatments.

Personalization: Tailored to the genetic profile of the patient’s cancer, enhancing the likelihood of a successful outcome.

Potential for Long-Term Results: May offer the possibility of long-term remission or cure by correcting underlying genetic issues.

Target Cancers

Gene Editing Therapy is primarily used to treat:

Hematologic cancers: Such as leukemia, lymphoma, and multiple myeloma.
Solid tumors: Including breast cancer, prostate cancer, and lung cancer.

Process

During Therapy

The therapy may involve outpatient procedures or hospital stays depending on the approach used.

After Therapy

Regular follow-up visits are necessary to monitor for side effects and assess the effectiveness of the treatment.

Recovery

Side effects and recovery experiences can vary; patients may experience temporary discomfort or immune-related reactions.

How is Gene Editing Therapy different from other therapies?

Gene Editing Therapy is unique because:

Targeted Approach: Directly addresses the genetic mutations driving cancer, unlike conventional therapies that may not specifically target these abnormalities.
Complex Procedure: Involves a complex process of stem cell collection, conditioning, and transplantation that requires specialized care.

Who is eligible for Gene Editing Therapy?

Eligibility depends on factors such as:

Type and stage of cancer
Overall health and medical history

A thorough evaluation by a healthcare team is necessary to determine if gene editing therapy is suitable for you.

How effective is this treatment?

Success rates can vary based on cancer type and individual patient factors. Clinical trials are ongoing to assess the effectiveness and safety of gene editing therapy, with promising results indicating its potential for significant impact.

Risks and side-effects

Off-Target Effects: Unintended changes to other parts of the genome could occur, potentially causing unwanted effects.
Immune Reactions: Possible immune responses against edited cells or therapy components.
Technical Challenges: The precision of gene editing must be carefully managed to avoid errors and ensure safety.

Next steps if Gene Editing Therapy doesn’t work

If gene editing therapy does not achieve the desired results, alternative options may include:

Other advanced therapies such as CAR-T or TCR therapy
Targeted therapies tailored to specific cancer characteristics
Participation in additional clinical trials
Supportive care to manage symptoms and improve quality of life

Insurance Coverage

Insurance coverage for gene editing therapy can vary widely by provider and plan. It is important for patients to consult with their healthcare provider and insurance company to understand their coverage options and potential costs.

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Therapies

What is Gene Editing Therapy?

How does Gene Editing Therapy work?

Benefits

Target Cancers

Process

During Therapy

After Therapy

Recovery

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