A Silver Bullet For Cancer Scientists Are Using Crispr To Unlock Patients True Tumor Fighting Potential

They modified patients’ genes to instruct cancer-fighting cells to swarm tumors using CRISPR, which is given as a single injection. CRISPR has previously been used in humans to remove specific genes to allow the immune system to become more active against cancer. But the new study was able to not only remove specific genes, but insert new ones that program immune cells to fight the patient’s own specific cancer. Dr Antoni Ribas, from the University of California, Los Angeles and co-leader of the study said: “This is a leap forward in the development of a personalized cancer treatment.” Scientists at pharmaceutical company PACT Pharma used gene-editing technology to isolate and clone cancer patients’ immune cells and prime them to target mutations in cancer cells. The researchers took blood and tumor samples from 16 patients with various types of cancer, including colon, breast and lung. They isolated immune system cells that had hundreds of mutations that specifically target the cancers plaguing their bodies. These were modified to be able to target each patient’s specific tumor, which has hundreds of unique mutations. One month after treatment, five of the participants had stable disease, meaning their tumors had not grown. The CRISPR tool consists of two main agents: a guide RNA and a DNA-cutting enzyme. A guide RNA is a specific RNA sequence that recognizes the target piece of DNA to be processed and directs the enzyme, Cas9, to begin the processing process. Cas9 precisely cuts the target DNA strands and removes a small piece, causing a gap in the DNA where a new piece of DNA can be added.

HOW DOES CRISPR WORK?

Crispr technology precisely changes small parts of the genetic code. Unlike other gene silencing tools, the Crispr system targets the starting material of the genome and permanently disables genes at the DNA level. DNA excision – known as double-strand break – closely mimics the kinds of mutations that occur naturally, for example after years of sun exposure. But unlike UV rays that can lead to genetic alterations, the Crispr system induces a mutation at a precise location in the genome. When the cellular machinery repairs the DNA break, it removes a small piece of DNA. In this way, researchers can precisely turn off specific genes in the genome. Scientists design the guide RNA to mirror the DNA of the gene to be edited, known as the target. The guide RNA works with the Cas9 enzyme and guides it to the target gene. When the guide RNA matches the DNA of the target gene, Cas9 cleaves the DNA, shutting down the target gene. Since the CRISPR technique has been around for about a decade, it remains the focus of ambitious scientific projects. Doctors are now investigating its application in the treatment of rare diseases and genetic disorders such as sickle cell disease. “The creation of a personalized cell therapy for cancer would not be possible without the new ability to use the CRISPR technique to replace immune receptors in clinical-grade cell preparations in a single step,” added Dr. Ribas. The findings give hope to the 1.9 million Americans who will be diagnosed with some form of cancer this year. About 290,000 women and 2,700 men will be diagnosed with breast cancer, making it the most common cancer diagnosis. Prostate cancer is the leading cancer diagnosis among men and the second most common diagnosis overall with approximately 269,000 cases expected this year. However, the technology is relatively new and raises some serious ethical questions about its application for genetic modification.
Medicine has entered uncharted territory in which hereditary disabilities could potentially be eliminated in a fetus. Safety issues in gene editing technology research are not unheard of. There is a risk of erroneously changing the DNA or RNA in regions other than the target site, which could lead to unwanted side effects not only in the patient but also in future generations. A major scandal rocked the world in 2019 when Chinese scientist He Jiankui was jailed after modifying the DNA of twin girls Lulu and Nana before birth to make them resistant to HIV. His work on manipulating the genes of human embryos was deemed “monstrous”, “unethical” and “highly dangerous”. A group of more than 100 scientists in China slammed He’s work in 2018: “Conducting direct experiments on humans can only be described as crazy.” The team added: “Pandora’s box has been opened. Maybe we still have a hope of closing it before it’s too late.’ In 2019, a group of scientists proposed a global moratorium on human germline processing. They wrote: “By ‘global moratorium’, we do not mean a permanent ban. Instead, we call for an international framework in which nations, while retaining the right to make their own decisions, voluntarily commit not to authorize any use of clinical germline processing unless certain conditions are met. PACT Pharma’s findings were published Thursday in the journal Nature.

title: “A Silver Bullet For Cancer Scientists Are Using Crispr To Unlock Patients True Tumor Fighting Potential " ShowToc: true date: “2022-11-24” author: “Ralph Phillips”

They modified patients’ genes to instruct cancer-fighting cells to swarm tumors using CRISPR, which is given as a single injection. CRISPR has previously been used in humans to remove specific genes to allow the immune system to become more active against cancer. But the new study was able to not only remove specific genes, but insert new ones that program immune cells to fight the patient’s own specific cancer. Dr Antoni Ribas, from the University of California, Los Angeles and co-leader of the study said: “This is a leap forward in the development of a personalized cancer treatment.” Scientists at pharmaceutical company PACT Pharma used gene-editing technology to isolate and clone cancer patients’ immune cells and prime them to target mutations in cancer cells. The researchers took blood and tumor samples from 16 patients with various types of cancer, including colon, breast and lung. They isolated immune system cells that had hundreds of mutations that specifically target the cancers plaguing their bodies. These were modified to be able to target each patient’s specific tumor, which has hundreds of unique mutations. One month after treatment, five of the participants had stable disease, meaning their tumors had not grown. The CRISPR tool consists of two main agents: a guide RNA and a DNA-cutting enzyme. A guide RNA is a specific RNA sequence that recognizes the target piece of DNA to be processed and directs the enzyme, Cas9, to begin the processing process. Cas9 precisely cuts the target DNA strands and removes a small piece, causing a gap in the DNA where a new piece of DNA can be added.

HOW DOES CRISPR WORK?

Crispr technology precisely changes small parts of the genetic code. Unlike other gene silencing tools, the Crispr system targets the starting material of the genome and permanently disables genes at the DNA level. DNA excision – known as double-strand break – closely mimics the kinds of mutations that occur naturally, for example after years of sun exposure. But unlike UV rays that can lead to genetic alterations, the Crispr system induces a mutation at a precise location in the genome. When the cellular machinery repairs the DNA break, it removes a small piece of DNA. In this way, researchers can precisely turn off specific genes in the genome. Scientists design the guide RNA to mirror the DNA of the gene to be edited, known as the target. The guide RNA works with the Cas9 enzyme and guides it to the target gene. When the guide RNA matches the DNA of the target gene, Cas9 cleaves the DNA, shutting down the target gene. Since the CRISPR technique has been around for about a decade, it remains the focus of ambitious scientific projects. Doctors are now investigating its application in the treatment of rare diseases and genetic disorders such as sickle cell disease. “The creation of a personalized cell therapy for cancer would not be possible without the new ability to use the CRISPR technique to replace immune receptors in clinical-grade cell preparations in a single step,” added Dr. Ribas. The findings give hope to the 1.9 million Americans who will be diagnosed with some form of cancer this year. About 290,000 women and 2,700 men will be diagnosed with breast cancer, making it the most common cancer diagnosis. Prostate cancer is the leading cancer diagnosis among men and the second most common diagnosis overall with approximately 269,000 cases expected this year. However, the technology is relatively new and raises some serious ethical questions about its application for genetic modification.
Medicine has entered uncharted territory in which hereditary disabilities could potentially be eliminated in a fetus. Safety issues in gene editing technology research are not unheard of. There is a risk of erroneously changing the DNA or RNA in regions other than the target site, which could lead to unwanted side effects not only in the patient but also in future generations. A major scandal rocked the world in 2019 when Chinese scientist He Jiankui was jailed after modifying the DNA of twin girls Lulu and Nana before birth to make them resistant to HIV. His work on manipulating the genes of human embryos was deemed “monstrous”, “unethical” and “highly dangerous”. A group of more than 100 scientists in China slammed He’s work in 2018: “Conducting direct experiments on humans can only be described as crazy.” The team added: “Pandora’s box has been opened. Maybe we still have a hope of closing it before it’s too late.’ In 2019, a group of scientists proposed a global moratorium on human germline processing. They wrote: “By ‘global moratorium’, we do not mean a permanent ban. Instead, we call for an international framework in which nations, while retaining the right to make their own decisions, voluntarily commit not to authorize any use of clinical germline processing unless certain conditions are met. PACT Pharma’s findings were published Thursday in the journal Nature.