Gene Editing to Enhance Human Embryos

MIT Technology Review notes that the cut-and-paste gene-editing technology CRISPR-Cas9 has the potential to treat and perhaps cure a great many diseases, but first one has to get CRISPR agents in the right place within the body. The article lists promising ways to do so, including gels and creams, drinkable or edible CRISPR, skin grafts, and ear injections, which seem especially promising to treat hereditary deafness.

Gene editing is prominently featured in last week’s bioscience news. CNN reports that scientists at the Francis Crick Institute have identified the role of a key gene that controls how embryos form during the first few days of development. The researchers used CRISPR-Cas9 to switch off a gene involved in embryo development, known as OCT4, thus halting the development of the embryo.

"We were surprised to see just how crucial this gene is for human embryo development, but we need to continue our work to confirm its role," said Norah Fogarty of the Francis Crick Institute, first author of the study, published in Nature.

The Guardian notes that the study, which is the first in the UK to have edited the DNA of human embryos, could lead to more effective fertility treatments, but “raises ethical questions about the prospect of controversial gene editing techniques being used clinically to correct defects in, or even enhance, human embryos in the future.”

Soft robotic actuator three times stronger than natural muscle. Researchers at Columbia Engineering have developed a 3D-printable synthetic soft muscle, a one-of-a-kind artificial active tissue with intrinsic expansion ability that does not require an external compressor or high voltage equipment as previous muscles required. In a study published in Nature Communications, the scientists show that the new material is three times stronger than natural muscle, and can lift 1000 times its own weight.

Gene therapy prevents and reverses multiple sclerosis in mice. Health scientists at University of Florida have found that multiple sclerosis can be inhibited or reversed using a novel gene therapy technique that stops the disease in laboratory mice. A research paper, published in Molecular Therapy, describes how the researchers were able to prevent the mouse version of multiple sclerosis by combining a brain-protein gene and an existing medication. The treatment produced near-complete remission in the animals, and the scientists are persuaded that the new technique has significant potential for treating multiple sclerosis and other autoimmune disorders.

Faster synthetic delivery channel for mRNA. MIT chemical engineers have designed a synthetic delivery system for Messenger RNA (mRNA), which carries genetic instructions from DNA to cell ribosomes and is considered to be a promising way to deliver therapeutic payloads because it degrades after being translated into the desired protein. A study published in Angewandte Chemie International Edition shows that the synthetic delivery system, which improve the rate of mRNA translation by attaching proteins to the ends of mRNA strands, is four times more effective than delivering mRNA on its own.

Kicking and killing HIV-infected cells. Scientists at UCLA and Stanford University have designed a synthetic molecule that can reactivate dormant human immunodeficiency virus (HIV) in mice and lead to the death of some of the infected cells. A study published in PLOS Pathogens describes a synthetic molecule that, imitating the molecule bryostatin 1, found in marine animals but difficult to extract, can trigger a "kick and kill" response.

Protein helps cells to choose the right way to repair DNA. Scientists at Salk Institute have discovered that a microprotein helps cells choose the best path to repair genes and avoid cancer. There are two pathways to repair genes, one faster and one slower but error-free, and the wrong choice could cause even more DNA damage and lead to cancer. The research work, published in Nature, reveals that a protein called CYREN helps cells choose the right pathway at the right time. According to the researchers, the discovery could open the door to new tools to fight cancer.

RNA microprograms instruct cells to efficiently fight disease. Scientists at the University of Warwick have found that cells can be programmed like a computer to fight cancer, influenza and other serious health conditions. A study published in Nucleic Acids Research describes how the researchers altered the gene expression of cells according to programs encoded in RNA, demonstrating that cells can be programmed with pre-defined RNA commands, like a computer’s microprocessor. According to the researchers, this is a synthetic biology breakthrough that could one day allow new personalized and efficient therapies based on instructing cells to execute complex actions encoded in RNA programs.