Gene Editing for Cell Therapy and Designer Babies

Scientists have found a way to efficiently and precisely remove genes from white blood cells of the immune system and to insert beneficial replacements, all in far less time than it normally takes to edit genes, New York Times reports. The researchers used the new method to repair a disease-causing genetic mutation in T cells from children with a rare genetic form of autoimmunity, and also created customized T cells to seek and kill human melanoma cells.

The new gene editing technique doesn’t use viruses to deliver DNA to cells (see below). “We needed something targeted, something fast and something efficient,” researcher Alex Marson told NYT. “What if we could just paste in a piece of DNA and avoid the viruses altogether?” CRISPR molecular “cut and paste” gene-editing technology permits avoiding viruses.

The researchers expect their technique to be widely adopted in the burgeoning field of cell therapy, accelerating the development of new and safer treatments for cancer, autoimmunity, and other diseases, including rare inherited disorders.

“This is a rapid, flexible method that can be used to alter, enhance, and reprogram T cells so we can give them the specificity we want to destroy cancer, recognize infections, or tamp down the excessive immune response seen in autoimmune disease,” said Marson. “Now we’re off to the races on all these fronts.”

Therapeutic genome editing research could prepare the way to heritable genome editing, which has recently been given a green light by UK bioethicists (see below). Inquiry Chair Karen Yeung said: "The implications for society are extensive, profound and long-term," as reported by BBC News.

Faster, cheaper, more precise CRISPR-based gene therapy without viruses. Scientists at UC San Francisco and collaborating labs have genetically reprogrammed the human immune cells known as T cells without using viruses to insert DNA. A research paper published in Nature describes a rapid, versatile, and economical approach based on CRISPR and applications of electric fields to cells to make their membranes temporarily more permeable. When certain quantities of T cells, DNA, and the CRISPR “scissors” are mixed together and then exposed to an appropriate electrical field, the T cells will take in these elements and integrate specified genetic sequences precisely at the site of a CRISPR-programmed cut in the genome.

Toward next-generation gene therapy platforms. Researchers at City of Hope have discovered a gene-editing technology that could efficiently and accurately correct the genetic defects that underlie certain diseases, positioning the new tool as the basis for the next generation of genetic therapies. A study published in PNAS describes a new genome editing platform, based on a family of non disease-causing viruses known as adeno-associated viruses, which may eventually be used to treat diseases such as sickle cell disease, hemophilia and other genetic disorders.

UK bioethicists give green light to heritable genome editing. An independent inquiry by the Nuffield Council on Bioethics, an independent body that examines and reports on ethical issues in biology and medicine, has concluded that editing the DNA of a human embryo, sperm, or egg to influence the characteristics of a future person ('heritable genome editing') could be morally permissible. If that is to happen, a number of measures would need to be put in place first to ensure that genome editing proceeds in ways that are ethically acceptable. The Council recommends the establishment of an independent body in the UK to promote broad and inclusive societal debate on heritable genome editing interventions and related scientific and medical developments.

Intelligent biohybrid materials for biotechnology and medicine. Scientists at the University of Freiburg have developed materials systems that are composed of biological components and polymer materials and are capable of perceiving and processing information. Three studies, published in Advanced Materials, Data in Brief, and Materials Today, describe biohybrid systems engineered to perform certain functions, such as counting signal pulses in order to release bioactive molecules or drugs at the correct time, or detecting enzymes and small molecules such as antibiotics in milk. The researchers are persuaded that these new materials might have broad applications in biotechnology and medicine.

Allergic responses may protect from skin cancer. Scientists at Imperial College London have suggested that the components of the immune system that trigger allergic reactions may also help protect the skin against cancer. The research results, published in Nature Immunology, suggest that exposure to environmental toxins and chemicals foreign to our body may trigger allergic responses, and that an antibody called Immunoglobulin E may have a crucial role in defending against the damage caused by environmental chemicals, and thereby protects against cancer.