A research team led by Prof. Gong Chen at Jinan University (Guangzhou, China) published a work on October 9th in Neural Regeneration Research, providing unambiguous data that brain internal astrocytes are directly converted into neurons through lineage tracing studies. Using brain internal astrocytes, a type of supporting cells to neurons, to directly convert into new neurons is an innovative brain repair technology that may benefit millions of patients worldwide.
Various genetic diseases caused by point mutations have no established therapeutic approaches. Prof. Tsukahara and colleagues (Japan Advanced Institute of Science and Technology) are studying a therapeutic method using artificial RNA editing.
A new study summarizes over 30 years of clinical experience in the treatment and management of glutaric acidemia type 1 (GA1), a rare and potentially devastating metabolic disorder caused by variants in the GCDH gene. The study followed the clinical course of 168 individuals with GA1 who were born between 1973 and 2019 and originated from 26 states and 6 countries.
University of Texas at Dallas researchers have taken an important step toward explaining why genetically identical cells can produce varying amounts of the same protein associated with the same gene. In a study published Aug. 18 and appearing in the Sept. 18 print edition of the journal Nucleic Acids Research, researchers demonstrated that most of the fluctuations in gene expression between identical cells occur in the first step of protein production, called transcription.
Scientists from an initiative launched by the Parker Institute for Cancer Immunotherapy (PICI) and the Cancer Research Institute called the Tumor Neoantigen Selection Alliance (TESLA) have discovered parameters to better predict which neoantigens can stimulate a cancer-killing effect.
The potential for treating cystic fibrosis (CF) using mRNA therapies or CRISPR gene editing is possible regardless of the causative mutation. CF clinical trials showing that a genotype-agnostic gene therapy for CF is possible
CNIO researchers destroyed Ewing's sarcoma and chronic myeloid leukaemia tumor cells by using CRISPR to cut out the fusion genes that cause them. For the first time, fusion genes have been selectively and efficiently removed using CRISPR. These genes are attracting a great deal of interest from the research community because they are unique to the tumor cell and are therefore excellent targets for the development of future drugs that only attack the tumor and are harmless for healthy cells.
Researchers develop a minimally invasive biodegradable microneedle patch as a novel delivery mode for gene therapy applications
Life-threatening cardiac arrhythmias can be linked to the functional and structural consequences of gene mutations.
New findings about body fat help explain the differing health risks men and women face - and set the stage for better, more targeted treatments.