Stem cells may offer hope for children with Hurler syndrome

November 16, 2017 by Lex Davis, University of Southern California
Stem cells may offer hope for children with Hurler syndrome
Toshio Miki’s stem cell research may help children born with Hurler syndrome. Credit: Cristy Lytal

A new therapy involving stem cells may offer a better quality of life for patients with Hurler syndrome, a rare genetic disease that can cause crippling physical conditions and lead to early death.

In work with mice, Toshio Miki of the Keck School of Medicine of USC and his team discovered that transplanting human placental can offer a long-term solutions to problems linked to the syndrome.

The disease leaves the body without a vital enzyme that breaks down large of bones and tissue. When these large molecules build up, they can damage organs and cause a host of problems, including a degeneration of brain functions, heart problems, impaired hearing and vision, loss of coordination, breathing difficulties, abnormal joint and bone growth, stiff joints, and a thickened skull and facial features.

Without or a blood or marrow transplant, children born with Hurler Syndrome usually die before they reach age 10.

In Miki's study, done with the support of the California Institute for Regenerative Medicine, transplanting healthy stem cells directly into the livers of newborn mice resulted in significant restored enzyme function for more than 20 weeks.

The mice that received stem-cell transplants had better coordination than those that didn't, and also had greatly reduced issues with bone development and joint stiffness.

Researchers even noticed that mice that received transplants were more likely to engage in normal grooming behavior and interact with their cage mates.

Miki's work is especially promising since it involves human placental stem cells, which are readily available.

Explore further: Stem cells hold hope for Hurler's syndrome

Related Stories

Stem cells hold hope for Hurler's syndrome

November 6, 2013
University of Adelaide research using special adult stem cells is promising new hope for better treatments for the devastating genetic disease Hurler's syndrome.

Engineered bone marrow could make transplants safer

May 8, 2017
Engineers at the University of California San Diego have developed biomimetic bone tissues that could one day provide new bone marrow for patients needing transplants.

Stem cell transplants may advance ALS treatment by repair of blood-spinal cord barrier

May 15, 2017
Researchers at the University of South Florida show in a new study that bone marrow stem cell transplants helped improve motor functions and nervous system conditions in mice with the disease Amyotrophic Lateral Sclerosis ...

Mouse study shows gene therapy may be possible cure for Hurler syndrome

February 4, 2014
Researchers used blood platelets and bone marrow cells to deliver potentially curative gene therapy to mouse models of the human genetic disorder Hurler syndrome – an often fatal condition that causes organ damage and other ...

New stem cell gene therapy gives hope to prevent inherited neurological disease

July 24, 2013
Scientists from The University of Manchester have used stem cell gene therapy to treat a fatal genetic brain disease in mice for the first time.

Mutations in bone cells can drive leukemia in neighboring stem cells

October 26, 2016
Certain DNA mutations in bone cells that support blood development can drive leukemia formation in nearby blood stem cells, researchers from Winship Cancer Institute, Emory University and Children's Healthcare of Atlanta ...

Recommended for you

New findings cast light on lymphatic system, key player in human health

October 16, 2018
Scientists at the Oklahoma Medical Research Foundation have broken new ground in understanding how the lymphatic system works, potentially opening the door for future therapies.

Age-related increase in estrogen may cause common men's hernia

October 16, 2018
An age-related increase in estrogen may be the culprit behind inguinal hernias, a condition common among elderly men that often requires corrective surgery, according to a Northwestern Medicine study was published Oct. 15 ...

New model suggests cuffless, non-invasive blood pressure monitoring possible using pulse waves

October 16, 2018
A large team of researchers from several institutions in China and the U.S. has developed a model that suggests it should be possible to create a cuffless, non-invasive blood pressure monitor based on measuring pulse waves. ...

Income and wealth affect the mental health of Australians, study shows

October 16, 2018
Australians who have higher incomes and greater wealth are more likely to experience better mental health throughout their lives, new research led by the Bankwest Curtin Economics Centre has found.

Discovery of inner ear function may improve diagnosis of hearing impairment

October 15, 2018
Results from a research study published in Nature Communications show how the inner ear processes speech, something that has until now been unknown. The authors of the report include researchers from Linköping University, ...

Widespread errors in 'proofreading' cause inherited blindness

October 12, 2018
Mistakes in "proofreading" the genetic code of retinal cells is the cause of a form of inherited blindness, retinitis pigmentosa (RP) caused by mutations in splicing factors.

0 comments

Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

Click here to reset your password.
Sign in to get notified via email when new comments are made.