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Gene-editing system targets multiple organs simultaneously
A gene-editing delivery system developed by UT Southwestern Medical Center researchers simultaneously targeted the liver and lungs of a preclinical model of a rare genetic disease known as alpha-1 antitrypsin deficiency (AATD), significantly improving symptoms for months after a single treatment, a new study shows.
Gene-editing nanoparticle system targets multiple organs simultaneously
A gene-editing delivery system developed by UT Southwestern Medical Center researchers simultaneously targeted the liver and lungs of a preclinical model of a rare genetic disease known as alpha-1 antitrypsin deficiency (AATD), significantly improving symptoms for months after a single treatment, a new study shows. The findings, published in Nature Biotechnology, could lead to new therapies for a variety of genetic diseases that affect multiple organs.
âMulti-organ diseases may need to be treated in more than one place. The development of multi-organ-targeted therapeutics opens the door to realizing those opportunities for this and other diseases,â said study leader Daniel Siegwart, Ph.D., Professor of Biomedical Engineering, Biochemistry, and in the Harold C. Simmons Comprehensive Cancer Center at UT Southwestern.
Gene editingâa group of technologies designed to correct disease-causing mutations in the genomeâhas the potential to revolutionize medicine, Dr. Siegwart explained. Targeting these technologies to specific organs, tissues, or cell populations will be necessary to effectively and safely treat patients.
Artificial neural networks reveal how peripersonal neurons represent the space around the body
The brains of humans and other primates are known to execute various sophisticated functions, one of which is the representation of the space immediately surrounding the body. This area, also sometimes referred to as âperipersonal space,â is where most interactions between people and their surrounding environment typically take place.
Researchers at Chinese Academy of Sciences, Italian Institute of Technology (IIT) and other institutes recently investigated the neural processes through which the brain represents the area around the body, using brain-inspired computational models. Their findings, published in Nature Neuroscience, suggest that receptive fields surrounding different parts of the body contribute to building a modular model of the space immediately surrounding a person or artificial intelligence (AI) agent.
âOur journey into this field began truly serendipitously, during unfunded experiments done purely out of curiosity,â Giandomenico Iannetti, senior author of the paper, told Medical Xpress. âWe discovered that the hand-blink reflex, which is evoked by electrically shocking the hand, was strongly modulated by the position of the hand with respect to the eye.
Herpes virus reshapes the human genome for its own benefit, but a single enzyme can stop it
Viruses are entirely dependent on their hosts to reproduce. They ransack living cells for parts and energy and hijack the hostâs cellular machinery to make new copies of themselves. Herpes simplex virus-1 (HSV-1), it turns out, also redecorates, according to a study in Nature Communications.
Researchers at the Center for Genomic Regulation (CRG) in Barcelona have discovered the cold sore virus reshapes the human genomeâs architecture, rearranging its shape in three-dimensional space so that HSV-1 can access host genes most useful for its ability to reproduce.
âHSV-1 is an opportunistic interior designer, reshaping the human genome with great precision and choosing which bits it comes into contact with. Itâs a novel mechanism of manipulation we didnât know the virus had to exploit host resources,â says Dr. Esther GonzĂĄlez Almela, first author of the study.