CRISPR is based on the biological system that some bacteria use to remove viruses from their DNA. CRISPR, along with the protein Cas9, a type of molecular scissors, has the ability to precisely locate a preprogrammed target DNA sequence, cut that sequence from the DNA and either allow the body to heal itself or a new genetically edited DNA sequence can be inserted to entirely wipe out a genetic condition. (Alice Park). In simple terms, the cells are removed, the DNA is altered, and genetically altered cells are reintroduced back into the body. This technology could entirely wipe out HIV, types of cancer, Sickle Cell anemia, Cystic Fibrosis, and many other genetic diseases. Using the CRISPR technology in drug therapy has scientists very excited. While drugs with this gene-editing technology are many years away, scientists are excited about the possibility of being able to go right to the root cause of a disease and repair a broken or mutated gene. Drugs based on the CRISPR-Cas9 combination have the ability to “complement the pills and biotech drugs that are currently available, targeting diseases that aren’t well treated by existing therapies. This is a new tool to target and treat disease” states Nessan Bermingham, CEO of Intellia Therapeutics, Inc (Rockoff). CRISPR technology is currently being used to genetically engineer mice and other animals so that they have human-like diseases that researchers can study. This technology is allowing the study of Amyotrophic Lateral sclerosis which, up until now, has lacked good animal models (Rockoff). Researchers, while excited about this breakthrough, are still trying to figure out the best way to deliver the drugs to the cells that are being targeted. Two
CRISPR is based on the biological system that some bacteria use to remove viruses from their DNA. CRISPR, along with the protein Cas9, a type of molecular scissors, has the ability to precisely locate a preprogrammed target DNA sequence, cut that sequence from the DNA and either allow the body to heal itself or a new genetically edited DNA sequence can be inserted to entirely wipe out a genetic condition. (Alice Park). In simple terms, the cells are removed, the DNA is altered, and genetically altered cells are reintroduced back into the body. This technology could entirely wipe out HIV, types of cancer, Sickle Cell anemia, Cystic Fibrosis, and many other genetic diseases. Using the CRISPR technology in drug therapy has scientists very excited. While drugs with this gene-editing technology are many years away, scientists are excited about the possibility of being able to go right to the root cause of a disease and repair a broken or mutated gene. Drugs based on the CRISPR-Cas9 combination have the ability to “complement the pills and biotech drugs that are currently available, targeting diseases that aren’t well treated by existing therapies. This is a new tool to target and treat disease” states Nessan Bermingham, CEO of Intellia Therapeutics, Inc (Rockoff). CRISPR technology is currently being used to genetically engineer mice and other animals so that they have human-like diseases that researchers can study. This technology is allowing the study of Amyotrophic Lateral sclerosis which, up until now, has lacked good animal models (Rockoff). Researchers, while excited about this breakthrough, are still trying to figure out the best way to deliver the drugs to the cells that are being targeted. Two