Sentences with phrase «targeting gene disorders»

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The technology's possibilities are staggering — in theory, allowing medical scientists to do everything from cure genetic disorders like sickle cell disease to identify gene targets for combating HIV.

That could eventually lead to new drugs that selectively target the gene, which McCarthy says, «could be a strategy for treating mood or sleep disorders.»

Yang said the study not only indicated which genes are affected by traumatic brain injury and linked to serious disease, but also might point to the genes that govern metabolism, cell communication and inflammation — which might make them the best targets for new treatments for brain disorders.

«By greatly narrowing the specific genes involved in social disorders, our findings will help uncover targets for treatment and provide measures by which these and other treatments are successful in alleviating the desperation of autism, anxiety and other disorders,» says Korenberg.

NIPD for single gene disorders in a fetus is diagnostic, as it targets specific genetic changes present in a high risk family.

However, this strong correlation between RORA and aromatase levels was not observed in females with autism spectrum disorder, suggesting that they may have compensatory mechanisms for regulating RORA target genes to offset RORA deficiency.

According to Sinclair, all of the mammalian SIRT genes (and their proteins) are possible drug targets for therapies aimed at extending life, as well as staving off age - related illnesses, such as Alzheimer's disease, cancers and metabolic disorders, like diabetes.

Gene therapy, which often employs viruses to deliver the good genes to a body's target cells, has been known to trigger severe immune responses and was blamed for the death of an 18 - year - old in 1999, who was receiving gene therapy for a hereditary metabolic disorGene therapy, which often employs viruses to deliver the good genes to a body's target cells, has been known to trigger severe immune responses and was blamed for the death of an 18 - year - old in 1999, who was receiving gene therapy for a hereditary metabolic disorgene therapy for a hereditary metabolic disorder.

Although variations in the NPAS1 and NPAS3 genes have been linked to brain disorders including autism, they aren't obvious drug targets because their function is most important in early life.

«A better understanding of the brain region and cell type - specific binding targets of Hnrnph1 will tell us more about the function of this gene and possibly identify new therapeutic strategies for minimizing risk and treating psychostimulant addiction — a disorder for which there is currently no FDA - approved drug,» explained corresponding author Camron Bryant, PhD, assistant professor of Pharmacology and Experimental Therapeutics & Psychiatry at BUSM.

Other blood disorders that have shown significant benefit from targeted gene therapy in small trials include hemophilia (specifically, factor IX deficiency), severe beta - thalassemia (deficiency for the adult beta - globin gene) and leukemia, where the patient's immune cells were treated to enable them to recognize cancer cells and destroy them.

Drug discovery for genetic diseases: Screening technologies are applied to identify chemical compounds able to inhibit REST function, a major target gene in Huntington's disease, and will serve as a paradigm for high - throughput screening approaches applied to neurological disorders.

This technology helps researchers connect mutations in the so - called genomic «dark matter» with the genes they affect, potentially revealing new therapeutic targets for genetic disorders.

Targeted gene therapy, which would affect smooth muscle cells but not skeletal muscle, holds promise for treatment of these and other disorders.

There are genes that, when mutated, cause disorders of language, speech and comprehension, and statistical analyses of our genomes show that these genes were targets of Darwinian natural selection.

Genes regulating the cerebral levels of important neurotransmitters (dopamine, serotonin, GABA, etc.) or signal transmission efficiency (neurotransmitter receptors and genes) have been targeted in association studies of major psychiatric disorders such as schizophrenia, bipolar disorders, attention deficit / hyperactivity disorder (ADHD), and autism [84], as well as of personality traits Genes regulating the cerebral levels of important neurotransmitters (dopamine, serotonin, GABA, etc.) or signal transmission efficiency (neurotransmitter receptors and genes) have been targeted in association studies of major psychiatric disorders such as schizophrenia, bipolar disorders, attention deficit / hyperactivity disorder (ADHD), and autism [84], as well as of personality traits genes) have been targeted in association studies of major psychiatric disorders such as schizophrenia, bipolar disorders, attention deficit / hyperactivity disorder (ADHD), and autism [84], as well as of personality traits [85].