Challenges of Gene Therapy Quiz

Challenges in Gene Therapy

• Gene therapy is a challenging field due to the difficulty of introducing new genes into cells and keeping them working.
• Delivering a normal gene to a large number of cells in the correct tissue and activating it to make the protein it encodes is crucial for gene therapy to work.
• Targeting the correct cells is important to avoid incorporating the therapeutic gene into the wrong cells, which could cause health problems for the patient or be passed on to their children.
• Gene-delivery vectors must avoid the body's natural surveillance system to prevent an immune response that could cause serious illness or death.
• The death of Jesse Gelsinger in a 1999 gene therapy trial sparked a discussion on how best to regulate experimental trials and report health problems in volunteer patients.
• To avoid triggering an immune response, researchers deliver viruses to cells outside the patient's body or give patients drugs to temporarily suppress the immune system during treatment.
• An introduced gene must become a permanent part of the target cell's genome for the therapy to last, but if it stitches itself into an inappropriate location, it could disrupt another gene and cause health problems.
• Gene therapy trials aimed at treating children with X-linked Severe Combined Immune Deficiency (SCID) resulted in some patients developing leukemia due to the newly transferred gamma c gene stitching itself into a gene that normally regulates cell division.
• Researchers have since developed safer ways to introduce genes, including using vectors that target DNA integration to specific "safe" places in the genome.
• Many genetic disorders that can potentially be treated with gene therapy are extremely rare, making it an unappealing prospect for pharmaceutical companies due to the high cost of development.
• Financial concerns raise questions about who will develop and pay for life-saving gene therapy treatments for rare disorders if companies cannot profit from them.
• Despite these challenges, gene therapy continues to evolve, and newer vectors with better targeting and integration features offer hope for future success in treating genetic disorders.

Challenges in Gene Therapy

• Gene therapy is a challenging field due to the difficulty of introducing new genes into cells and keeping them working.
• Delivering a normal gene to a large number of cells in the correct tissue and activating it to make the protein it encodes is crucial for gene therapy to work.
• Targeting the correct cells is important to avoid incorporating the therapeutic gene into the wrong cells, which could cause health problems for the patient or be passed on to their children.
• Gene-delivery vectors must avoid the body's natural surveillance system to prevent an immune response that could cause serious illness or death.
• The death of Jesse Gelsinger in a 1999 gene therapy trial sparked a discussion on how best to regulate experimental trials and report health problems in volunteer patients.
• To avoid triggering an immune response, researchers deliver viruses to cells outside the patient's body or give patients drugs to temporarily suppress the immune system during treatment.
• An introduced gene must become a permanent part of the target cell's genome for the therapy to last, but if it stitches itself into an inappropriate location, it could disrupt another gene and cause health problems.
• Gene therapy trials aimed at treating children with X-linked Severe Combined Immune Deficiency (SCID) resulted in some patients developing leukemia due to the newly transferred gamma c gene stitching itself into a gene that normally regulates cell division.
• Researchers have since developed safer ways to introduce genes, including using vectors that target DNA integration to specific "safe" places in the genome.
• Many genetic disorders that can potentially be treated with gene therapy are extremely rare, making it an unappealing prospect for pharmaceutical companies due to the high cost of development.
• Financial concerns raise questions about who will develop and pay for life-saving gene therapy treatments for rare disorders if companies cannot profit from them.
• Despite these challenges, gene therapy continues to evolve, and newer vectors with better targeting and integration features offer hope for future success in treating genetic disorders.