CRISPR-based therapies are rapidly advancing, with applications involving cell replacement therapies, gene drives, and diagnostics. The use of CRISPR both ex vivo and in vivo is hindered by the immunogenicity of CRISPR components, limited delivery, low specificity, off-target effects, DNA damage, and repair pathways.
What are some challenges of gene editing?
- Gene therapy is not a new field; it has been evolving for decades. Despite the best efforts of researchers around the world, however, gene therapy has seen only limited success.
- Gene delivery and activation.
- Immune response.
- Disrupting important genes in target cells.
- Commercial viability.
What are problems with gene editing?
A lab experiment aimed at fixing defective DNA in human embryos shows what can go wrong with this type of gene editing and why leading scientists say it's too unsafe to try. In more than half of the cases, the editing caused unintended changes, such as loss of an entire chromosome or big chunks of it.
What are the disadvantages of gene editing?
- Potential unintended, or "off-target," effects.
- Increased likelihood of developing cancer.
- Possibility of being used in biological attacks.
- Unintended consequences for future generations.
What are 3 challenges of gene therapy?
- Unwanted immune system reaction. Your body's immune system may see the newly introduced viruses as intruders and attack them.
- Targeting the wrong cells.
- Infection caused by the virus.
- Possibility of causing a tumor.