Leukodystrophies News and Research

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Gene therapy's double-edged sword: Breakthrough treatments face manufacturing and efficacy

Research explores the advancements, challenges, and economic implications of gene therapy for various diseases, including inherited blood disorders, malignancies treated with CAR-T cells, and diseases targeted by in vivo AAV vectors. The review highlights the transformative potential of gene therapies while acknowledging the complexities in manufacturing, safety, efficacy, and long-term durability.

12 Nov 2023

How do low levels of oxygen damage the brain?

In this interview, Professor Paul Tesar and Kevin Allan speak to News-Medical about how low levels of oxygen damage the brain.

6 Nov 2020

Umbilical cord blood safely and effectively treats children with rare genetic disorders

Researchers at UPMC Children's Hospital of Pittsburgh found that infusing umbilical cord blood - a readily available source of stem cells - safely and effectively treated 44 children born with various non-cancerous genetic disorders, including sickle cell, thalassemia, Hunter syndrome, Krabbe disease, metachromatic leukodystrophy (MLD) and an array of immune deficiencies.

14 Jul 2020

Researchers make breakthrough in understanding cause of rare childhood brain disorders

A new breakthrough in understanding the cause of rare childhood brain disorders has been made by researchers at the University of Sheffield.

27 Apr 2020

Mimicking Human Brain Development Using Organoids

Dr. Paul Tesar from the Tesar Laboratory at Case Western University, Ohio, discusses the importance of organoids in biological research and the development of organoids which are capable of simulating the early stages of human myelin.

7 Sep 2018

Researchers use novel PET tracer to assess myelin damage in mouse models of MS

The loss or damage of myelin, a cellular sheath that surrounds and insulates nerves, is the hallmark of the immune-mediated neurological disorder multiple sclerosis.

12 Jan 2018

Wayne State researchers receive $1.9 million NIH grant to better understand rare genetic disorders

A team of Wayne State University researchers recently received a $1.9 million grant from the National Eye Institute of the National Institutes of Health to better understand leukodystrophies (LD) and genetic Leukoencephalopathies (gLE), rare genetic disorders affecting the white matter -- myelin -- in the central nervous system.

21 Jul 2017

Ubiquitous human herpesvirus 6 may play key role in brain diseases like multiple sclerosis

The ubiquitous human herpesvirus 6 (HHV-6) may play a critical role in impeding the brain's ability to repair itself in diseases like multiple sclerosis.

10 Jul 2017

Rat study reveals new clues to devastating neurological disease

After a 29-year quest, Ian Duncan, a professor of veterinary medicine at the University of Wisconsin-Madison, has finally pinpointed the cause of a serious neurologic disease in a colony of rats.

20 Apr 2017

Scientists use non-invasive way to track rapid myelination of nerve fibers in children's brains

Much like electricity traveling down wires, nerve impulses in our brain travel along nerve fibers. And just as wires need insulation to function well, nerve fibers, too, rely on a kind of insulation called myelin, a fatty substance that protects them and increases the speed at which nerve impulses travel.

6 May 2016

International research team identifies new gene associated with 4H leukodystrophy

Leukodystrophies are deadly neurodegenerative diseases that affect one in 7,000 children and remain incurable. These genetic diseases attack myelin or the "insulating rubber sheath" surrounding neurons, which leads to deteriorating health for affected children.

9 Jul 2015

UC Davis researchers settle long-standing controversy surrounding Canavan disease

UC Davis investigators have settled a long-standing controversy surrounding the molecular basis of an inherited disorder that historically affected Ashkenazi Jews from Eastern Europe but now also arises in other populations of Semitic descent, particularly families from Saudi Arabia.

28 Apr 2015

Scientists develop method to generate functioning brain cells that produce myelin

Stem cell technology has long offered the hope of regenerating tissue to repair broken or damaged neural tissue. Findings from a team of UC Davis investigators have brought this dream a step closer by developing a method to generate functioning brain cells that produce myelin - a fatty, insulating sheath essential to normal neural conduction.

29 Aug 2013

Drug used to control Type II diabetes can help repair spinal cords of mice suffering from inherited disease

A drug used to control Type II diabetes can help repair the spinal cords of mice suffering from the inherited disease adrenoleukodystrophy which, untreated, leads eventually to a paralysis, a vegetative state and death.

10 Jun 2013

UC Davis scientists detect novel molecular target for multiple sclerosis

Working with lab mice models of multiple sclerosis, UC Davis scientists have detected a novel molecular target for the design of drugs that could be safer and more effective than current FDA-approved medications against MS.

17 May 2013

New treatment strategy for patients with CMT disease on the horizon

A potential new treatment strategy for patients with Charcot-Marie-Tooth disease is on the horizon, thanks to research by neuroscientists now at the University at Buffalo's Hunter James Kelly Research Institute and their colleagues in Italy and England.

29 Apr 2013

Researchers transform skin cells directly into oligodendrocyte precursor cells

Researchers at the Stanford University School of Medicine have succeeded in transforming skin cells directly into oligodendrocyte precursor cells, the cells that wrap nerve cells in the insulating myelin sheaths that help nerve signals propagate.

16 Apr 2013

Researchers discover new technique that directly converts skin cells into functional brain cells

Researchers at Case Western Reserve School of Medicine have discovered a technique that directly converts skin cells to the type of brain cells destroyed in patients with multiple sclerosis, cerebral palsy and other so-called myelin disorders.

15 Apr 2013

Reprogrammed human skin cells highly effective in treating myelin disorders

A study out today in the journal Cell Stem Cell shows that human brain cells created by reprogramming skin cells are highly effective in treating myelin disorders, a family of diseases that includes multiple sclerosis and rare childhood disorders called pediatric leukodystrophies.

10 Feb 2013

Researchers unlock complex cellular mechanics that instruct glial progenitor cells to divide

In a new study appearing this month in the Journal of Neuroscience, researchers have unlocked the complex cellular mechanics that instruct specific brain cells to continue to divide. This discovery overcomes a significant technical hurdle to potential human stem cell therapies; ensuring that an abundant supply of cells is available to study and ultimately treat people with diseases.

2 Nov 2012