Gene Therapy News and Research

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Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery.

Researchers create cellular and spatial “atlas” of the choroid plexus during different life stages

Once viewed merely as a producer of the cerebrospinal fluid (CSF) bathing the brain and spinal cord, the choroid plexus is now known to be a key player in brain development and immunity.

7 Jun 2021

A lack of oversight for unproven stem cell interventions is a global public health problem

Authors are urging the WHO to establish an Exert Advisory Committee on Regenerative Medicine to provide harmony to the national regulations on the SCI industry.

4 Jun 2021

Researchers develop proof-of-concept treatment for blood disorders

Researchers at Children's Hospital of Philadelphia (CHOP) have developed a proof-of-concept treatment for blood disorders like sickle cell disease and beta-thalassemia that could raise hemoglobin levels by activating production of both fetal and adult hemoglobin.

1 Jun 2021

UVA researchers shed new light on brain development

University of Virginia School of Medicine researchers have shed new light on how our brains develop, revealing that the very last step in cell division is crucial for the brain to reach its proper size and function.

31 May 2021

Study sheds new light on human brain development

29 May 2021

Researchers to conduct Phase 1 gene therapy trial to treat Alzheimer's disease

The National Institute on Aging (NIA), part of the National Institutes of Health, has awarded a $5 million grant to researchers at University of California San Diego School of Medicine to conduct a first-in-human Phase 1 clinical trial of a gene therapy for treating Alzheimer's disease (AD) or Mild Cognitive Impairment (MCI), a condition that often precedes full-blown dementia.

26 May 2021

Researchers identify mechanism that may lead to new therapies for strokes, brain injuries

In a surprising discovery, researchers at Massachusetts General Hospital (MGH) identified a mechanism that protects the brain from the effects of hypoxia, a potentially lethal deprivation of oxygen.

26 May 2021

Scientists discover key component to repair the molecular motor of human heart

A team of scientists from Loyola University Chicago's Stritch School of Medicine have discovered a critical component for renewing the heart's molecular motor, which breaks down in heart failure.

20 May 2021

Study could serve as guidepost for developing new therapies for Alzheimer's disease

In a major breakthrough, researchers at Massachusetts General Hospital (MGH) have discovered how amyloid beta--the neurotoxin believed to be at the root of Alzheimer's disease (AD)--forms in axons and related structures that connect neurons in the brain, where it causes the most damage.

20 May 2021

Researchers find new clue to restore congenital hearing loss

Researchers at Oregon State University have found a key new piece of the puzzle in the quest to use gene therapy to enable people born deaf to hear.

19 May 2021

Scientists develop experimental direct-acting antiviral therapy to treat COVID-19

An international team of scientists from the Menzies Health Institute Queensland (MHIQ) at Griffith University and from City of Hope, a research and treatment center for cancer, diabetes and other life-threatening diseases in the U.S., have developed an experimental direct-acting antiviral therapy to treat COVID-19.

18 May 2021

UNSW researchers find novel way to attack the 'fortress' surrounding pancreatic tumors

Tackling the scar tissue that shields pancreatic tumors from effective drug access is a promising advance in a notoriously hard-to-treat cancer.

17 May 2021

Nanotechnology and COVID-19 diagnosis and treatment

The development of nano-biosensors and nanoparticle-based vaccines and medicines has opened a new path toward better management of the coronavirus disease 2019 (COVID-19) pandemic. In a recently published article in the journal ACS Biomaterials Science & Engineering, scientists have reviewed recent advancements in nanotechnology-based diagnostic and therapeutic interventions against human coronaviruses.

17 May 2021

Investigational gene therapy can restore immune function in children with inherited immunodeficiency disorder

An investigational gene therapy can safely restore the immune systems of infants and children who have a rare, life-threatening inherited immunodeficiency disorder, according to research supported in part by the National Institutes of Health.

12 May 2021

Personalized vaccine can provide effective immunization against cancer

Marie-Claude Bourgeois-Daigneault and her team at the CRCHUM are using mice to show how a combination of peptides and oncolytic viruses, used as an adjuvant, can provide effective immunization against cancer.

11 May 2021

Novel 'CopyCatcher' detects when precise copying of genetic information takes place in cells

Researchers at the University of California San Diego have laid the groundwork for a potential new type of gene therapy using novel CRISPR-based techniques.

11 May 2021

Experimental gene therapy successfully treats children born without an immune system

An experimental form of gene therapy developed by a team of researchers from UCLA and Great Ormond Street Hospital in London has successfully treated 48 of 50 children born with a rare and deadly inherited disorder that leaves them without an immune system.

11 May 2021

Absence of key gene causes severe hearing loss

A gene called GAS2 plays a key role in normal hearing, and its absence causes severe hearing loss, according to a study led by researchers in the Perelman School of Medicine at the University of Pennsylvania.

7 May 2021

Nanoscope presents optical gene delivery, OCT-guided electrophysiology platforms at ARVO annual meeting

Nanoscope Technologies LLC, a biotechnology company developing gene therapies for treatment of retinal diseases, is featuring multiple scientific presentations highlighting its groundbreaking research on optical gene delivery for vision restoration and OCT-guided electrophysiology platforms for characterization of retinal degeneration and assessment of efficacy of cell-gene therapy at the 2021 ARVO annual (virtual) meeting, May 1-7.

5 May 2021

Researchers use gene therapy to prevent learning, memory loss in Alzheimer's mouse model

Researchers at University of California San Diego School of Medicine, with colleagues elsewhere, have used gene therapy to prevent learning and memory loss in a mouse model of Alzheimer's disease (AD), a key step toward eventually testing the approach in humans with the neurodegenerative disease.

5 May 2021

Gene Therapy News and Research

Further Reading

Researchers create cellular and spatial “atlas” of the choroid plexus during different life stages

A lack of oversight for unproven stem cell interventions is a global public health problem

Researchers develop proof-of-concept treatment for blood disorders

UVA researchers shed new light on brain development

Study sheds new light on human brain development

Researchers to conduct Phase 1 gene therapy trial to treat Alzheimer's disease

Researchers identify mechanism that may lead to new therapies for strokes, brain injuries

Scientists discover key component to repair the molecular motor of human heart

Study could serve as guidepost for developing new therapies for Alzheimer's disease

Researchers find new clue to restore congenital hearing loss

Scientists develop experimental direct-acting antiviral therapy to treat COVID-19

UNSW researchers find novel way to attack the 'fortress' surrounding pancreatic tumors

Nanotechnology and COVID-19 diagnosis and treatment

Investigational gene therapy can restore immune function in children with inherited immunodeficiency disorder

Personalized vaccine can provide effective immunization against cancer

Novel 'CopyCatcher' detects when precise copying of genetic information takes place in cells

Experimental gene therapy successfully treats children born without an immune system

Absence of key gene causes severe hearing loss

Nanoscope presents optical gene delivery, OCT-guided electrophysiology platforms at ARVO annual meeting

Researchers use gene therapy to prevent learning, memory loss in Alzheimer's mouse model

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