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CRISPR Gene Editing Cures Inherited Disorders

15 June 2020
Giulio Prisco


Blood transfusions are normally used to treat severe forms of beta thalassaemia and sickle cell disease. However, three people with these inherited diseases no longer require transfusions. Their bone marrow stem cells were gene-edited with CRISPR, New Scientist reports.

A recent trial was the first to use CRISPR to treat inherited genetic disorders. And results were announced at a virtual meeting of the European Hematology Association.

“I am encouraged by the preliminary results, which demonstrate, in essence, a functional cure for patients with beta thalassemia and sickle cell disease,” said senior researcher Haydar Frangoul. The statement was in a press release issued by CRISPR Therapeutics and Vertex Pharmaceuticals Incorporated.

Artificial Eyes May Provide Superhuman Vision

Scientists led by Hong Kong University of Science and Technology have developed the world's first 3D artificial eye. It has capabilities better than existing bionic eyes. And, in some cases, it even exceeds those of natural human eyes. This brings vision to humanoid robots and new hope to patients with visual impairment.

The Electrochemical Eye (EC-Eye) is described in a research paper published in Nature. It replicates the structure of a natural eye. And it could offer sharper vision than a human eye in the future, with extra functions such as the ability to detect infrared radiation in darkness.

The key feature allowing such breakthroughs is a 3D artificial retina. It's made of an array of nanowire light sensors that mimic the photoreceptors in human retinas.

The Importance of a Strategic Mindset

Psychologists at National University of Singapore have suggested that one important psychological factor behind the achievements of successful people may be a "strategic mindset." This is the attitude of those who, in the face of challenges or setbacks, ask themselves: "How else can I do this? Is there a better way of doing this?"

A study published in PNAS suggests that a strategic mindset can be taught.

Light-Activated Molecules Kill Tumors

Chemists at Rice University have turned fluorescent tags into cancer killers. Switching one atom in the tag does the trick.

A research paper published in Chemical Science reports that replacing a single oxygen atom with a sulfur atom in a common fluorophore turns it into a photosensitizing molecule. When exposed to light, the molecule generated reactive oxygen species (ROS) that destroyed breast cancer cells in the lab.

Cheaper Compounds with Anti-Cancer Effects

Researchers at Tokyo University of Science have developed a method through which a fungal compound, capable of activating the self-destruct gene in certain cancer cells, can be artificially produced in marketable quantities. This provides a potential cancer therapeutic strategy.

In a paper published in European Journal of Organic Chemistry, the researchers describe a method to produce a compound with known anti-cancer properties. It is called "FE399." And it is naturally found in Ascochyta, a species of filamentous fungus.

According to the researchers, the newly produced compound could provide an unprecedented treatment option for patients with colorectal cancer.

Vitamin D Can Protect Against Certain Cancers

Scientists at University of Eastern Finland and Autonomous University of Madrid have found that vitamin D is beneficial both in cancer prevention and in the prognosis of several cancers.

A research paper published in Seminars in Cancer Biology indicates that the anti-cancer effects of vitamin D are especially pronounced in the prevention and treatment of colon cancer and blood cancers.

In addition, high vitamin D responsiveness can be linked to a smaller cancer risk. Vitamin D responsiveness varies between individuals, affecting their need for vitamin D supplementation.

Wireless Neural Stimulation for Neural Conditions

Rice University neuroengineers have created a tiny surgical implant that can electrically stimulate the brain and nervous system without using a battery or wired power supply.

The new neural stimulator is described in a study published in Neuron. It is based on a thin film of "magnetoelectric" material that converts magnetic energy directly into an electrical voltage.

The device worked in laboratory mice that were fully awake and free to roam. It could be used to treat epilepsy, Parkinson's disease, chronic pain, and other conditions.

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