Gene Therapy and Cell Therapy

Gene Therapy

• The main goal of gene replacement is to replace a faulty or missing gene with a healthy copy.
• The main goal of gene editing is to correct a specific mutation or defect in a gene.
• Gene suppression is used to reduce or eliminate the expression of a specific gene.

Gene Transfer

• The ideal vector for gene transfer is typically a viral vector, as it can efficiently deliver genes to target cells.
• Lentivirus is a type of vector that can integrate into a site-specific location.
• Retroviral vectors are useful for treating genetic disorders, but may have a potential risk of insertional mutagenesis.

Cell Therapy

• Ex vivo therapy involves removing cells from the body, modifying them in a lab, and then re-introducing them into the body.
• This type of cell therapy is often used in gene therapy to modify cells before re-introducing them into the body.

Vector Characteristics

• The ability of a vector to integrate into a site-specific location determines the transient or permanence of expression in gene therapy.
• 3rd generation lentiviruses are designed to be safer and more efficient, with a characteristic of having a self-inactivating (SIN) design.

Gene Therapy Concerns

• A concern related to the use of gene therapies in general is the potential for off-target effects or unintended consequences.

Monoclonal Antibodies (mAbs)

• A disadvantage of monoclonal antibodies (mAbs) is that they may not be able to penetrate solid tumors.
• The primary obstacle in using monoclonal antibodies (mAbs) for cancer treatment is the development of resistance.
• The main application of Western blot using antibodies is to detect and analyze specific proteins in a sample.
• Trastuzumab (Herceptin) is a monoclonal antibody that targets the HER2 protein in cancer treatment.
• Gemtuzumab (Mylotarg) is a monoclonal antibody conjugated to the cytotoxic agent calicheamicin, used to treat acute myelogenous leukemia.