Latest Research and Innovations in Genetics: Summer 2019

New medical technologies and innovations have greatly enhanced the how scientists are able to work with genetics. Gene therapy treatments, analysis of fully sequenced genomes, and simplified genetic tests are just a few examples of how researchers are leveraging new capabilities to improve genetic healthcare. Below, DocWire News has compiled the most recent and significant headlines regarding medical innovations in genetic technologies.

LEAPER: New Genetic Editing Approach May Rival CRISPR

Scientists from Peking University have recently created a gene-editing technique that has great potential in treating disease. This technique, known as LEAPER, is similar to CRISPR-Cas13 in its ability to edit RNA rather than DNA; however, the Peking researchers feel that LEAPER presents several advantages over the CRISPR-Cas13 technique. Their findings were published recently in Nature Biotechnology.

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Study Finds Gene Therapy Effective in Treating Thalassemia

Research presented last week at the 24th Congress of European Hematology Association (EHA) in Amsterdam found gene therapy to be effective in treating animal models of thalassemia. By targeting genes associated with iron regulation and erythropoiesis, or the production of red blood cells, they were able to successfully dampen the symptoms of the blood disorder. These findings were presented by Dr. Antonella Nai of the San Raffaele Scientific Institute in Milan, Italy.

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Is Genetically Editing Mosquitoes the Best Way to Fight Malaria?

Malaria presents as one of the leading causes of disease and death in many developing countries. In 2016, the CDC estimates the disease to have accounted for 216 million cases, resulting in 445,000 deaths. The need for effective malaria treatments is eminent, but it is a particularly challenging disease to create vaccines or therapies for. One novel approach to this issue is not to treat or vaccinate humans, but rather to make mosquitoes invincible to malaria.

This approach revolves around the concept of gene drive, a genetic engineering technique that quickly spreads a specific gene throughout an entire population. Scientists have been working to create gene drives in insects for decades, however once CRISPR-Cas9 emerged, efforts towards gene drive in mosquitoes really took off. CRISPR-Cas9 is a gene editing tool that allows researchers to cut DNA and paste in genetic material of their choosing, creating genetically modified organisms.

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Using Light to Change Genetic Activity in the Brain

Researchers from the University at Buffalo recently managed to control a gene that is essential in the human growth process using light. This innovative work was done in lab-grown brain tissue and has the potential to create new cancer treatments and manage mental disorders like schizophrenia, the authors say. This work was led by UB researchers Josep M. Jornet, Michal K. Stachowiak, Yongho Bae and Ewa K. Stachowiak, and was published in Proceedings of the Institute of Electrical and Electronics Engineers.

The researchers note that this approach is pushing forward gene manipulation, posing potential treatments for various diseases. The approach focuses on controlling human genes using nanotechnology and laser lights, a technique they are calling optogenomics.

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Researchers Create Gene Therapy for Arrhythmia Using a Stem Cell Model

A research team from the Boston Children’s Hospital recently created the first tissue model of an inherited heart arrhythmia and treated it with gene therapy in an animal model. This work not only provides more information regarding the condition but supports gene therapy as a one dose treatment for patients with the disease. These findings were published in two separate papers in the journal Circulation.

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