CRISPR Technology Offers New Hope for Patients with Devastating Diseases

Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) is a powerful new gene-editing tool with unprecedented implications for the future of medicine, agriculture, energy, and indeed, humanity.

Key Highlights

With CRISPR technology at the forefront of scientific developments, we discuss the potential this tool has for clinical research and as a treatment for patients.

Samantha Mineroff
Samantha Mineroff

The discovery of the CRISPR-Cas microbial adaptive immune system and its ongoing development into a genome-editing tool represents the work of many scientists from around the world. Scientists such as Francisco Mojica, Virginijus Siksnys, and Feng Zhang all contributed to CRISPR’s development, which culminated most recently with the awarding of the Nobel Prize for Chemistry to Emmanuelle Charpentier and Jennifer Doudna for their work on its development.

CRISPR’s beginnings go back to 1987 when Japanese scientists studying E. coli noticed unusual repeating sequences in the bacteria’s DNA. In time, researchers discovered that the sequences were part of the bacteria’s immune system, which produced a special attack enzyme—such as Cas9—to chop up the attacker’s DNA and neutralize the threat. Thus, the idea of using CRISPR as a gene-editing tool was born.

“CRISPR’s use in developing animal models as compared to previous methods is an important step in preclinical development,” explains says Amy Raymond, PhD, Director of Therapeutic Expertise, Center for Rare Diseases. “Anywhere we can introduce efficiency into the overall drug discovery and development process is a step closer to getting experimental medicines into trials and the complete data package to regulators for review earlier. If proven safe and effective, this would be treatment to patients sooner. Patients are waiting—there is no time to waste at any step.”

This genome tool essentially harnesses components of the bacterial or archaeal immune system to edit genes in other organisms like plants, animals, and people. This is a unique mechanism of CRISPR, but genome-editing is not limited to this technology alone. Other genome-editing tools include transcription activator-like effector nucleases (TALENs), zinc-finger nucleases (ZFNs), and homing endonucleases. While these tools have existed for years, these methods are costly, requiring cumbersome machines and materials. CRISPR’s potential is far greater than anything else to date. CRISPR could be a much more efficient and precise form of genetic surgery.

“CRISPR technology, in terms of logistics, precision, and elegant operation, is where we're all looking for durable solutions with respect to genetically-driven diseases,” says Dr. Raymond. “CRISPR-mediated treatments could bring relief to cancer and rare diseases patients, changing the course for these devastating illnesses.”

CRISPR-mediated treatments could bring relief to cancer and rare diseases patients, changing the course for these devastating illnesses.

Amy Raymond, PhD, Director of Therapeutic Expertise, Center for Rare Diseases

CRISPR shows potential in vaccine development as well. While pharma scientists have long used bacteria, yeast, and mammal cells to develop vaccines, they are now looking to plants or plant cells to help with “molecular pharming.” CRISPR could help precisely insert specific genes into plants, allowing researchers to study how plant genes are regulated, how they respond to foreign molecules, and how they repair their DNA. CRISPR could then be used to create vaccine drug products for humans that can illicit immunogenicity against infectious diseases or cancer biomarkers in a cancer therapeutic vaccine approach. Such new knowledge may lead to vaccines that have previously been difficult to develop and manufacture.

CRISPR’s impact on medicine will likely be transformative for treatment options. Already it’s been shown to edit bone marrow cells in mice to treat sickle-cell anemia, and CRISPR-mediated treatments are being tested in human clinical trials now. Researchers have also been exploring its use in destroying DNA viruses such as HIV, herpes, HPV, and hepatitis. CRISPR might even stop such genetic diseases as Huntington’s disease or cystic fibrosis, and could be the essential tool in one day creating powerful and much-needed new antibiotics.

CRISPR is also used for some genetically modified cellular therapies, such as introducing chimeric antigen receptors (CARs) into somatic cells either from a patient or donor, making what have become revolutionary therapies. This and other CRISPR derived technologies—such as ssDNA CRISPR—are cost-effective editing technologies that have been transformative in creation of CAR-T therapies, both in the treatment of oncology/hematology indications and even some non-malignant indications. We are finally able to talk about truly curative potential for diseases like cancer.

We will continue to follow this incredible technology with optimism as we look to continue supporting our partners in developing therapeutics and treatments for patients across the world.

Learn more about our Center for Immuno-oncology, Cellular, and Gene Therapy.

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