Lifeboat Foundation

Just seven years after scientists announced the first use of Crispr-Cas9 gene editing technology on human cells, researchers shared new evidence this week that Crispr can be used to cure two serious genetic disorders.

On Tuesday, NPR reported that a patient in Nashville had seen a dramatic decline in her symptoms of sickle cell disease after receiving a single gene therapy treatment in July. Sickle cell, which can lead to inflammation, debilitating pain, and life-threatening circulatory problems, affects millions of people around the world.

That same day, the biotech companies behind the sickle-cell treatment, Crispr Therapeutics and Vertex, also shared promising results from their first attempt to cure a case of beta thalassemia, another genetic disorder that affects blood proteins. Nine months after receiving the experimental treatment, a patient in Germany with beta thalassemia has almost no signs of the disorder.

Promising preliminary data from one of the first human trials testing the safety and efficacy of a CRISPR gene therapy has just been revealed. Although it is too early to evaluate long-term effects, the initial reports are impressively successful for two patients with severe genetic blood diseases.

Until February of this year, when pharmaceutical companies CRISPR Therapeutics and Vertex began a large global trial into a treatment called CTX001, no human outside of China had been officially treated with a CRISPR-based gene editing therapy.

CTX001 was developed to treat two types of inherited blood disease, beta-thalassemia and sickle cell disease. Both conditions are caused by a mutation in a single gene and the treatment involves engineering a patient’s stem cells with a single genetic change designed to raise levels of fetal hemoglobin in red blood cells.

The city of the future might produce the humans of it, too.

When an undiagnosed rare genetic disease caused his young son’s kidneys to fail, Professor Chris Toumazou vowed to find a way of uncovering hidden health risks.

The professor of biomedical engineering realised that, although his son’s condition could not have been prevented, the family could have managed his lifestyle very differently had they known about his condition.

Continue reading “This wristband tells you what food to buy based on your DNA” »

Doctors have reported on the first attempts in the United States to use gene editing to help patients fight cancer.

The doctors say one form of gene editing appeared to be safe when tested in three patients. But it is not yet known what long-term effects the method will have on cancer treatment or patient survival rates.

A gene editing tool called CRISPR/Cas9 was used in the tests, which were recently reported in a medical study. The method was discovered in recent years as a way to change the genetic material that make up a person’s DNA.

To successfully engineer cell or tissue implants, bioengineers must facilitate their metabolic requirements through vascular regeneration. However, it is challenging to develop a broad strategy for stable and functional vascularization. In a recent report on Nature Communications, Wei Song and colleagues in the interdisciplinary departments of Biological and Environmental Engineering, Medicine, Mechanical and Aerospace Engineering, Clinical Sciences and Bioengineering in the U.S. described highly organized, biomimetic and resilient microvascular meshes. The team engineered them using controllable, anchored self-assembly methods to form microvascular meshes that are almost defect-free and transferrable to diverse substrates, for transplantation.

The scientists promoted the formation of functional blood vessels with a density as high as ~200 vessels per mm^-2 within the subcutaneous space of SCID-Beige mice. They demonstrated the possibility of engineering microvascular meshes using human induced pluripotent stem-cell (iPSCs) derived endothelial cells (ECs). The technique opens a way to engineer patient-specific type 1 diabetes treatment by combining microvascular meshes for subcutaneous transplantation of rat islets in SCID-beige mice to achieve correction of chemically induced diabetes for 3 months.

Vasculature is an essential component of any organ or tissue, and vascular regeneration is critical to successfully bioengineer implants. For instance, during cell replacement therapy for type 1 diabetes (T1D), transplanted insulin producing cells rely on the vasculature to function and survive. Bioengineers often use vascular endothelial cells such as human umbilical vein endothelial cells (HUVECs) to spontaneously assemble into tubular structures within the extracellular matrix (ECM). But the resulting structures can be random, uncontrollable and less efficient for microvascular regeneration. Scientists have recently developed three-dimensional (3D) printing techniques to engineer controlled cellular constructs with embedded vessels. However, it remains challenging to 3D print resilient and transferrable, high-resolution, microvasculature.

Prime Editing, A New Gene Editing Technique May Offer Improvement Over CRISPR : Shots — Health News A new technique, dubbed ‘prime editing,’ appears to make it even easier to make very precise changes in DNA. It’s designed to overcome the limits of the CRISPR gene editing tool.