Gene News and Research

Latest Gene News and Research

Scientists use AHDS patients’ cells to recreate disease and mimic blood-brain barrier

The blood-brain barrier is biology's proverbial double-edged sword. An impermeable shield of endothelial cells that protects our brains from toxins and other threats that may lurk in circulating blood, the barrier can also exclude therapeutic drugs and, at times, essential biomolecules required for healthy brain development.

17 May 2017

Researchers discover likely cause for visual impairment in Finnish variant of NCL

Researchers from the University of Eastern Finland have discovered a likely cause for visual impairment and eventual loss of vision in the Finnish variant of Neuronal Ceroid Lipofuscinosis.

17 May 2017

Spanish scientists develop promising gene therapy method to treat Wilson's disease

Scientists at the Center for Applied Medical Research at the University of Navarra have designed a promising gene therapy method to treat Wilson's disease, a rare pathology caused by the lack of or malfunction of a gene.

16 May 2017

Researchers identify many dangerous strains of TB pathogens

Researchers from the Vavilov Institute of General Genetics of the Russian Academy of Sciences (VIGG) and the Moscow Institute of Physics and Technology have established a catalog of mutations in 319 virulence genes of mycobacteria that cause tuberculosis.

16 May 2017

Study unravels new clues to cause and treatment of spinal muscular atrophy

Spinal muscular atrophy, a neurodegenerative disease that causes progressive muscle wasting and paralysis, may be partly due to abnormalities in the synapses that connect sensory neurons and motor neurons, according to researchers at Columbia University Medical Center.

16 May 2017

JAX researcher awarded federal grant for genomic studies of healthy aging and Alzheimer's disease

A grant totaling $2,668,127 over five years will fund studies of the complex processes involved in both healthy aging and Alzheimer's disease, in the laboratory of Jackson Laboratory (JAX) Assistant Professor Catherine Kaczorowski, Ph.D.

16 May 2017

Composition, function of intestinal bacteria could predict response to biologic drugs in IBD patients

Analysis of a patient's gut microbiome - the microbial population of the gastrointestinal tract - may be able to predict the likelihood of successful treatment for inflammatory bowel disease with biologic drugs that target immune system activity.

16 May 2017

IBM scientists identify new way to trigger the body's immune response to fight diseases

IBM announced its researchers have identified a new way to trigger the body's immune response by using polymer-coated graphene sheets. The research was recently published in Nature Communications.

16 May 2017

Diabetes medication could potentially help treat symptoms of Fragile X syndrome

Metformin, the most widely used drug to treat type 2 diabetes, could potentially be used to treat symptoms of Fragile X syndrome, an inherited form of intellectual disability and a cause of some forms of autism.

15 May 2017

New gene variant linked to Lou Gehrig's disease

For the first time, a variant in UBQLN4 gene has been associated with Lou Gehrig's disease or amyotrophic lateral sclerosis (ALS) - a progressive disease resulting in the loss of nerve cells that control muscle movement, which eventually leads to paralysis and death.

15 May 2017

Discovery of steroid hormone in fruit flies may shed new light on maternal hypothyroidism

University of Oregon researchers using Drosophila have identified a steroid hormone that triggers a vital transition in early brain development in which neural stem cells properly change gears to produce different kinds of neurons.

15 May 2017

Research networks focus on investigating spread of antimicrobial resistance in humans, animals

"Antimicrobial-resistant bacteria pose a complex challenge. This is why Germany, with its German Antimicrobial Resistance Strategy DART2020, is making sustained efforts to protect the health of humans and animals", says BfR President Professor Dr. Dr. Andreas Hensel.

12 May 2017

Phase 1 clinical studies reveal safety, tolerability of therapeutic vaccine in celiac disease patients

ImmusanT, Inc., a clinical-stage company developing Nexvax2, a therapeutic vaccine intended to protect against the effects of gluten exposure while maintaining a gluten-free diet in HLA-DQ2.5+ patients with celiac disease, today announced the publication of positive data from two Phase 1 clinical trials of Nexvax2 in celiac disease.

12 May 2017

New spherical nucleic acid drug crosses blood-brain barrier to target brain tumors in animals

The first drug using spherical nucleic acids to be systemically given to humans has been developed by Northwestern University scientists and approved by the Food and Drug Administration as an investigational new drug for an early-stage clinical trial in the deadly brain cancer glioblastoma multiforme.

12 May 2017

Analyzing single-cell RNA data in pre-malignant tumors

RNA sequencing is basically the isolation of RNA from cells and the use of reverse transcriptase to turn the RNA into DNA. You can then use your standard DNA sequencing technologies to quantify the cDNA that you obtained from the reverse transcription reaction.

12 May 2017

Researchers uncover structural details that make eye-targeting virus a better tool for future therapies

In their quest to replicate themselves, viruses have gotten awfully good at tricking human cells into pumping out viral proteins. That's why scientists have been working to use viruses as forces for good: to deliver useful genes to human cells and help patients who lack important proteins or enzymes.

12 May 2017

Research findings identify protein as potential new drug target for HCMV infections

Scientists have demonstrated that a human protein known as valosin containing protein (VCP) is essential for replication of human cytomegalovirus. The findings, published in PLOS Pathogens, identify VCP as a potential new treatment target.

11 May 2017

Preclinical data reinforces potential efficacy, durability of investigational gene therapy for DMD

Solid Biosciences announced today that new data from two preclinical studies reinforce the potential of its investigational microdystrophin gene therapy, SGT-001, to be an effective treatment approach for Duchenne muscular dystrophy.

11 May 2017

LMU scientists uncover molecular mechanism that links tumor hypoxia and metastasis

The low oxygen concentrations that prevail in many tumors enhance their propensity to metastasize to other tissues. Researchers at Ludwig-Maximilians-Universitaet in Munich led by Professor Heiko Hermeking have now uncovered the molecular mechanism that links the two phenomena.

11 May 2017

CUMC researchers create new organoids that mimic features of full-sized lung

New lung "organoids"-tiny 3-D structures that mimic features of a full-sized lung- have been created from human pluripotent stem cells by researchers at Columbia University Medical Center. The team used the organoids to generate models of human lung diseases in a lab dish, which could be used to advance our understanding of a variety of respiratory diseases.

11 May 2017

Gene News and Research

Latest Gene News and Research

Scientists use AHDS patients’ cells to recreate disease and mimic blood-brain barrier

Researchers discover likely cause for visual impairment in Finnish variant of NCL

Spanish scientists develop promising gene therapy method to treat Wilson's disease

Researchers identify many dangerous strains of TB pathogens

Study unravels new clues to cause and treatment of spinal muscular atrophy

JAX researcher awarded federal grant for genomic studies of healthy aging and Alzheimer's disease

Composition, function of intestinal bacteria could predict response to biologic drugs in IBD patients

IBM scientists identify new way to trigger the body's immune response to fight diseases

Diabetes medication could potentially help treat symptoms of Fragile X syndrome

New gene variant linked to Lou Gehrig's disease

Discovery of steroid hormone in fruit flies may shed new light on maternal hypothyroidism

Research networks focus on investigating spread of antimicrobial resistance in humans, animals

Phase 1 clinical studies reveal safety, tolerability of therapeutic vaccine in celiac disease patients

New spherical nucleic acid drug crosses blood-brain barrier to target brain tumors in animals

Analyzing single-cell RNA data in pre-malignant tumors

Researchers uncover structural details that make eye-targeting virus a better tool for future therapies

Research findings identify protein as potential new drug target for HCMV infections

Preclinical data reinforces potential efficacy, durability of investigational gene therapy for DMD

LMU scientists uncover molecular mechanism that links tumor hypoxia and metastasis

CUMC researchers create new organoids that mimic features of full-sized lung

Industry focus eBook - Cell and gene therapy (2nd edition) eBook

Advancing brain microphysiological systems (bMPS)

PhD researcher at LifeArc

Unveiling Hidden Potential: Organoids for Disease Modeling in Neuroscience Research