611 W. Union Street
Benson, AZ 85602
(520) 586-0800

Health Choice Integrated Care crisis Line
1-877-756-4090

NurseWise 24-Hour Crisis Line
1-866-495-6735

NAZCARE Warm Line
1-888-404-5530



SEABHS
611 W. Union Street
Benson, AZ 85602
(520) 586-0800

NurseWise 24-Hr Crisis Line
1-866-495-6735

NAZCARE Warm Line
1-888-404-5530


powered by centersite dot net

Getting Started
Here are some forms to get started. These can be printed and brought with you so that you can pre-fill out some known info ahead of time. More...


Health Sciences
Resources
Basic InformationLatest News
Gene Tweaking Prevented Obesity in MiceApproach That New Gene Testing Kit With CautionResearch on Almost 2,000 Brains Brings Insight Into Mental IllnessRestoring Hair Growth on Scarred Skin? Mouse Study Could Show the WayParkinson's Gene Therapy Wires New Brain CircuitsNext for Disabling Back Pain? New Discs From Patients' Own CellsSkin 'Glow' Test Might Someday Spot Disease Risk EarlyComputer-Brain Link Helps 'Locked In' People Chat, Surf WebCould a Natural Protein Help Fight Obesity?Blood Test May One Day Help Track Concussion RecoveryThe Bigger the Brain, the Bigger the Tumor Risk?Gene Therapy for Parkinson's Symptoms Shows PromiseCould Same-Sex Couples Have Babies With Shared DNA? Study Hints It's PossibleMany Americans Curious, But Wary, About Gene TestingAHA: New Report Explores Genes Behind Congenital Heart DiseaseScientists Find 500 More Genes That Influence Blood PressureALS Affects the Mind, Not Just the BodyScientists Finally Get Around to Finding Procrastination's Home in the BrainGene 'Editing' in Dog Study Shows Promise for Kids With Muscular DystrophyGut Enzyme Could Help Solve U.S. Blood Shortages'Fat' Mouse Test Failure Yields New Obesity ClueIs Evolution of the Human Brain to Blame for Some Mental Disorders?Scientists Trace Link Between Head Injuries and Parkinson'sAHA: Scientists May Have Cleared Gene Therapy HurdleAlmost 1,300 Genes Seem Tied to Academic SuccessBrains May Be as Unique as Fingerprints'Heading' Soccer Balls May Be Bad for BalanceScientists Target Cellular 'Fountain of Youth' to Extend Mouse Life SpanThose At-Home DNA Tests Are an Imperfect ScienceScientists Spot Gene Linking Down Syndrome, Early Alzheimer'sMassive Study Finds Same Genes Drive Many Psychiatric ConditionsThyroid Cancer Survivors at Risk for Heart DiseaseBetter Diet, Bigger Brain?Primary Care Providers Have Mixed Views on Genetic TestsFDA Targets Clinics Offering Unapproved Stem Cell TherapiesRestless Legs Linked to Brain ChangesContact Sports May Alter the Brain, Scans SuggestJust One Concussion Could Raise Parkinson's RiskLove Your Hair Color? You Have Over 100 Genes to Thank.Too Much Sitting Could Raise Brain RisksBusting Myths Surrounding Cancer and Genetic TestingTough Times Can Leave Their Mark on the Older BrainSugar-Craving Gene Helps Lower Body Fat, But Has DownsideMajor Project Completes Genetic 'Map' of 33 CancersOlder Brains Replenish Cells Just Like Young Brains: StudyScientists Say They Discovered a 'New Organ' in the BodyNew Technology Gives 'Feeling' to Prosthetic ArmsBlood Pressure Check? There May Soon Be an App for ThatHealth Tip: What You Can Learn From Genetic TestingAs Stroke 'Liquefies' Brain Tissue, Lasting Harm May Spread
Questions and AnswersLinksBook Reviews
Related Topics

Medical Disorders
Mental Disorders
Mental Health Professions

Gene 'Editing' in Dog Study Shows Promise for Kids With Muscular Dystrophy

HealthDay News
by By Amy NortonHealthDay Reporter
Updated: Aug 30th 2018

new article illustration

THURSDAY, Aug. 30, 2018 (HealthDay News) -- For the first time, scientists report using gene-editing technology to halt the progression of muscular dystrophy in dogs -- suggesting a possible breakthrough for children with a form of the disease.

Reporting in the current issue of Science, researchers describe how they used CRISPR technology to edit a naturally occurring genetic flaw that causes a version of Duchenne muscular dystrophy in dogs. CRISPR is an acronym for a family of DNA sequences.

The gene correction, in turn, triggered what the scientists call an "unprecedented" improvement in the animals' muscle fibers.

The discovery might hold the key to helping children with Duchenne MD, the most common form of muscular dystrophy. MD is a group of incurable genetic disorders that cause progressive muscle degeneration.

Duchenne MD primarily affects boys and usually arises in early childhood. Historically, most boys did not survive beyond their teens, but more are living into their 30s these days, according to the Muscular Dystrophy Association.

Duchenne is caused by a mutation in a gene that produces a critical protein called dystrophin. Without it, muscles throughout the body -- including the heart and diaphragm -- break down over time.

"The only real way to correct this disorder is to get the body to produce functional dystrophin," said Eric Olson, lead researcher on the new study. He's professor and director of University of Texas Southwestern Medical Center's Hamon Center for Regenerative Science.

To do that, researchers have been studying gene therapy. But with Duchenne MD, Olson said, there is a hurdle to replacing the defective gene with a functioning one: its size.

"It's simply too massive to replace," he explained.

So Olson and his team took a different approach. They used CRISPR gene-editing technology to fix the gene defect.

The researchers treated four dogs that carried the most common mutation seen in people with Duchenne MD -- affecting a location on the dystrophin gene called exon 51. They used a harmless virus to deliver CRISPR components to the exon -- which the technology then "edited."

Within weeks, the researchers reported, the missing dystrophin protein was restored in muscle throughout the animals' bodies.

The effects were not uniform. In some muscle, dystrophin was produced at 3 percent of its normal level. But in the heart and diaphragm, the protein was restored to 92 percent and 58 percent of normal, respectively.

The researchers also found evidence of improved integrity in the animals' muscle fibers.

Olson put the increases in dystrophin levels in perspective: There is a drug for Duchenne MD -- called Exondys 51, and approved in the United States in 2016 -- that can be used in a minority of patients who have a mutation in exon 51.

It has been shown to restore less than 1 percent of dystrophin in skeletal muscle after one year, Olson pointed out.

"Here, we saw really dramatic changes -- beyond what we'd hoped for," he said.

The findings are "very encouraging," said Dr. Sajel Lala Kana, a clinical geneticist at Nicklaus Children's Hospital in Miami.

But they are also very early, she pointed out. "This study shows what happens in these animals in the short term," said Kana, who was not involved in the research. "But will this be sustainable over a long period of time?"

Olson agreed that that is a critical question. And then there's the issue of whether the gene editing could have unintended adverse effects.

There are two main theoretical safety concerns, according to Olson: "Are there any off-target effects?" he said. "That is, could this accidentally affect the expression of other genes?"

Another question, Olson said, is whether the immune system will react to the enzyme CRISPR uses to make its gene fixes. So far, there have been no signs of that, the researchers said.

While animal studies frequently don't produce the same results in humans, Olson said these findings can be seen as a promising early step.

"We're working hard on trying to treat the cause of this disease," he said. "With further study in animals, in a few years we may be able to move into human trials."

Kana agreed. "There's a lot of research going on this field," she said. "This is how we'll move toward a cure someday."

In the United States, Duchenne MD affects one in every 3,500 to 6,000 boys born each year, according to the National Institutes of Health. Often, the agency says, there is no family history of the disease; instead, mutations spontaneously occur in the dystrophin gene.

More information

The Muscular Dystrophy Association has more on Duchenne muscular dystrophy.