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AAV-based gene therapy to treat liver disorders advances into human testing

AAV-based gene therapy to treat liver disorders advances into human testing

Liver-directed gene therapy delivered using adeno-associated viral (AAV) vectors to treat diseases such as hemophilia have advanced into human testing. [More]
Studies in non-human primates provide more predictive results in genetic causes of blindness

Studies in non-human primates provide more predictive results in genetic causes of blindness

Many gene therapy-based approaches are in development to combat genetic and other causes of blindness and vision loss, and much can be learned about the safety and effectiveness of these promising new therapies by studying them first in non-human primates before initiating clinical trials, as shown by the results of a study published in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. [More]
Ionis announces FDA approval of first SMA drug in the U.S for pediatric and adult patients

Ionis announces FDA approval of first SMA drug in the U.S for pediatric and adult patients

Ionis Pharmaceuticals, Inc. announced today that the U.S. Food and Drug Administration has approved SPINRAZATM (nusinersen) under Priority Review for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients. [More]
Scientists discover OGDHL and NRD1 genes linked to progressive loss of neurological function

Scientists discover OGDHL and NRD1 genes linked to progressive loss of neurological function

An international team of scientists has discovered that the gene, OGDHL, a key protein required for normal function of the mitochondria -- the energy-producing factory of the cell -- and its chaperone, nardilysin (NRD1) are linked to progressive loss of neurological function in humans. [More]
Wellcome Trust Sanger Institute establishes spin-out company, Microbiotica

Wellcome Trust Sanger Institute establishes spin-out company, Microbiotica

Cambridge Innovation Capital plc, a Cambridge-based investor in technology and healthcare companies, and IP Group plc, the developer of intellectual property-based businesses, announce the creation of Microbiotica Ltd, a newly formed spin-out company from the UK’s Wellcome Trust Sanger Institute based at the Wellcome Genome Campus in Hinxton, Cambridge. [More]
Innovation in stroke aftercare across Europe: an interview with Professor Urs Fischer

Innovation in stroke aftercare across Europe: an interview with Professor Urs Fischer

Stroke is the epidemic disease of the twenty-first century and the second most frequent cause of death in 2011, accounting for 11% of all deaths worldwide. Stroke is also the second most important cause of permanent disability and... [More]
MGH researchers develop magnetic coils for selective and reliable neural stimulation

MGH researchers develop magnetic coils for selective and reliable neural stimulation

Massachusetts General Hospital researchers have developed what appears to be a significant improvement in the technology behind brain implants used to activate neural circuits responsible for vision, hearing or movement. [More]
NIH scientists unravel chain of events that lead to fatal outcomes in cerebral malaria

NIH scientists unravel chain of events that lead to fatal outcomes in cerebral malaria

Using state-of-the-art brain imaging technology, scientists at the National Institutes of Health filmed what happens in the brains of mice that developed cerebral malaria (CM). [More]
OHSU scientists identify potential target for preventing and treating Alzheimer's disease

OHSU scientists identify potential target for preventing and treating Alzheimer's disease

A new scientific discovery may provide a future avenue for treatment and prevention of Alzheimer's disease. [More]
Aerobic activity offers greater preservation of brain volume, improves cognitive performance

Aerobic activity offers greater preservation of brain volume, improves cognitive performance

Using a new MRI technique, researchers found that adults with mild cognitive impairment (MCI) who exercised four times a week over a six-month period experienced an increase in brain volume in specific, or local, areas of the brain, but adults who participated in aerobic exercise experienced greater gains than those who just stretched. [More]
Recessive gene mutations could cause debilitating neurological disease in children

Recessive gene mutations could cause debilitating neurological disease in children

Researchers have linked a debilitating neurological disease in children to mutations in a gene that regulates neuronal development through control of protein movement within neuronal cells. [More]
Researchers develop new mouse model to study Zika virus and find potential treatments

Researchers develop new mouse model to study Zika virus and find potential treatments

Researchers have developed a new mouse model that could be used in Zika research to better understand the virus and find new treatments, according to a study published in PLOS Pathogens. [More]
New mouse models may open ways to develop therapies for ALS patients

New mouse models may open ways to develop therapies for ALS patients

Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease, is a progressive, fatal neurological disease that attacks the nerve cells controlling voluntary muscles. No effective treatments have been found. [More]
Stem cell therapy appears to dampen the body's neuroinflammatory response to trauma, preserve brain tissue

Stem cell therapy appears to dampen the body's neuroinflammatory response to trauma, preserve brain tissue

Results of a cellular therapy clinical trial for traumatic brain injury (TBI) using a patient's own stem cells showed that the therapy appears to dampen the body's neuroinflammatory response to trauma and preserve brain tissue, according to researchers at The University of Texas Health Science Center at Houston. [More]
New review highlights promises and challenges of nanotechnology in neuroscience

New review highlights promises and challenges of nanotechnology in neuroscience

Despite the numerous challenges associated with the application of nanotechnology in neuroscience, it promises to have a significant impact on our understanding of how the nervous system works, how it fails in disease, and the development of earlier and less-invasive diagnostic procedures so we can intervene in the pre-clinical stage of neurological disease before extensive neurological damage has taken place. [More]
New bionic hybrid neuro chip records brain cell activity at higher resolution

New bionic hybrid neuro chip records brain cell activity at higher resolution

Brain functions are controlled by millions of brain cells. However, in order to understand how the brain controls functions, such as simple reflexes or learning and memory, we must be able to record the activity of large networks and groups of neurons. [More]
Rare neurological syndromes linked to variations in ATAD3A gene

Rare neurological syndromes linked to variations in ATAD3A gene

A team of scientists from a number of institutions around the world, including Baylor College of Medicine, has discovered that rare neurological syndromes for which there was no cause can be the result of variations in the gene ATAD3A. [More]
UA neuroscientists receive $10.3 million grant to find cause for heightened risk of Alzheimer's in women

UA neuroscientists receive $10.3 million grant to find cause for heightened risk of Alzheimer's in women

Why do more women than men get Alzheimer's disease? In their quest to find the answer, neuroscientist Roberta Diaz Brinton, PhD, and her collegues in the Center for Innovation in Brain Science at the University of Arizona Health Sciences, have been awarded a $10.3 million five-year Program Project Grant from the National Institute on Aging at the National Institutes of Health. [More]
Cedars-Sinai receives FDA approval to examine safety of combination stem cell-gene therapy in ALS patients

Cedars-Sinai receives FDA approval to examine safety of combination stem cell-gene therapy in ALS patients

Cedars-Sinai regenerative medicine investigators have received approval from the U.S. Food and Drug Administration to test a combination stem cell-gene therapy they developed to stall the progression of amyotrophic lateral sclerosis, a neurological disease that causes progressive paralysis and ultimately death. [More]
Researchers discover how toxic proteins linked to neurological diseases impair membrane-less organelles

Researchers discover how toxic proteins linked to neurological diseases impair membrane-less organelles

Researchers at St. Jude Children's Research Hospital have discovered the way toxic proteins linked to the most common forms of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) incapacitate membrane-less organelles inside cells. [More]
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