Breaking News
April 21, 2019 - More TV, Tablets, More Attention Issues at Age 5
April 21, 2019 - Drug reduces risk of kidney failure in people with diabetes, study finds
April 21, 2019 - New research identifies novel link between antibiotic resistance and climate change
April 21, 2019 - Simple intervention can provide lasting protection for teens against junk food marketing
April 21, 2019 - The protein p38-gamma identified as a new therapeutic target in liver cancer
April 21, 2019 - Novel system enables researchers to study bacteria within mini-tissues in a dish
April 21, 2019 - Discovery of oral cancer biomarkers could save thousands of lives
April 21, 2019 - Geneva Exhibition committee gives gold medals to two medications developed by Kazan
April 21, 2019 - Scientists aim to minimize or eliminate hair loss during cancer treatment
April 21, 2019 - WiFi interacts with signaling pathways in the human brain
April 21, 2019 - Stroke Hospitalizations Down in Black, White Medicare Enrollees
April 21, 2019 - First common risk genes discovered for autism
April 21, 2019 - Researchers map auditory sensory system of the mouse brain
April 21, 2019 - Scientists Bring Pig’s Brain, Dead 4 Hours, Back to ‘Cellular Activity’
April 21, 2019 - Virtual reality a promising tool for reducing fears and phobia in autism
April 21, 2019 - New analysis lists out opportunities for U.S. medical schools to advance population health
April 21, 2019 - More sleep may help teens with ADHD focus and organize
April 21, 2019 - Breakthrough antibody treatment suppresses HIV without antivirals
April 21, 2019 - AveXis Data Reinforce Effectiveness of Zolgensma in Treating Spinal Muscular Atrophy (SMA) Type 1
April 21, 2019 - Is your hand pain arthritis, carpal tunnel or something else?
April 21, 2019 - Measles outbreaks may become more frequent if vaccination rates continue to decline
April 21, 2019 - Researchers succeed in accelerating process of creating 3D images
April 21, 2019 - Tiny worm mimics key genetic risk for Alzheimer’s
April 21, 2019 - Angry dreams explained by brain waves
April 20, 2019 - Parenteral Antimicrobial Tx at Home Burdens Children’s Caregivers
April 20, 2019 - Diabetes treatment may keep dementia, Alzheimer’s at bay
April 20, 2019 - New bandage-like biosensor collects and analyzes sweat
April 20, 2019 - A comprehensive, centralized database of bovine milk compounds
April 20, 2019 - Two new epigenetic regulators maintain self-renewal of embryonic stem cells
April 20, 2019 - New Evidence That Veggies Beat Steak for Heart Health
April 20, 2019 - Study reveals genes associated with heavy drinking and alcoholism
April 20, 2019 - Texas A&M AgriLife becomes the newest member of NutriRECS international consortium
April 20, 2019 - In most states, insurance won’t cover addiction treatments
April 20, 2019 - Computer-based memory games may be beneficial for individuals with fragile X syndrome
April 20, 2019 - Timing of food intake influences molecular clock in the liver of mice
April 20, 2019 - Precise decoding of breast cancer cells paves way for new treatment option
April 20, 2019 - Scientists use 3D imaging to help model complex processes performed by placenta
April 20, 2019 - MediciNova Announces Plans to Move Forward with a Phase 3 Trial of MN-166 (ibudilast) in ALS
April 20, 2019 - Genetic variants that protect against obesity could aid new weight loss medicines
April 20, 2019 - New technology developed for microscopic imaging in living organisms
April 20, 2019 - when quitting cigarettes, consider using more nicotine, not less
April 20, 2019 - Key proteins can block Listeria without triggering the death of host cells
April 20, 2019 - Researchers create a working model of cerebral tract to study brain function
April 20, 2019 - New study shows that microbes can help break toxic chemical in dust
April 20, 2019 - Scientists use NIR light and injected DNA nanodevice to guide stem cells to injury
April 20, 2019 - Microbial Features ID’d for Pediatric Irritable Bowel Syndrome
April 20, 2019 - Study reveals patterns of drug intoxication deaths, organ donors across the US
April 20, 2019 - Scientists deploy CRISPR gene-editing tool to engineer multiple edits
April 20, 2019 - AHA News: Here’s How Middle-Aged People — Especially Women — Can Avoid a Heart Attack
April 20, 2019 - Charcot foot: MedlinePlus Medical Encyclopedia
April 20, 2019 - France to ban popular breast implants over cancer risk: media
April 20, 2019 - Researchers explore whether time of day can affect the body’s response to physical exertion
April 20, 2019 - CPAP brings longer life for obese people with sleep apnea: Study
April 20, 2019 - New discovery transforms conventional microfluidics into open-space microfluidics
April 20, 2019 - An accurate estimation of the overall cost of bacterial resistance in French hospitals during 2015 and 2016
April 20, 2019 - ‘PRO-cision Medicine’ approach helps personalize care for patients with cancer
April 19, 2019 - TG Therapeutics Receives Orphan Drug Designation for Umbralisib from the U.S. Food and Drug Administration for the Treatment of Marginal Zone Lymphoma
April 19, 2019 - Screen time—even before bed—has little impact on teen well-being: study
April 19, 2019 - Cytosurge’s first FluidFM User Conference
April 19, 2019 - New study finds that previously described differences among endoscopists are not true
April 19, 2019 - Study compares effectiveness and cost of gene therapy and HSCT in major beta-thalassemia
April 19, 2019 - Scientific breakthrough provides new hope for people living with multiple sclerosis
April 19, 2019 - New Virtual Reality Therapy game could offer relief for patients with chronic pain, mobility issues
April 19, 2019 - Emergency medicine doctors find better way to treat severe epileptic seizures in children
April 19, 2019 - MedlinePlus: Cholesterol Good and Bad
April 19, 2019 - For busy medical students, two-hour meditation study may be as beneficial as longer course
April 19, 2019 - Music therapy helps young patients feel better
April 19, 2019 - Molecular target UNC45A is essential for cancer cell proliferation and tumor growth
April 19, 2019 - Crackling and wheezing could be the sounds of a progressing lung disease
April 19, 2019 - Key research takeaways from ECCMID 2019
April 19, 2019 - AI Can Identify Model of Cardiac Rhythm Device From Chest X-Ray
April 19, 2019 - New way to combat childhood anxiety: treat the parents
April 19, 2019 - Women getting C-sections best judge of own pain medication needs | News Center
April 19, 2019 - Light-intensity physical activity associated with healthy brain aging
April 19, 2019 - Immune responses that prevent fungal infections may eliminate Trichinella spiralis
April 19, 2019 - Exercising in the morning, rather than at night, may yield better results, shows study
April 19, 2019 - Why eating ‘right’ could cause you to stray from your diet
April 19, 2019 - Health Tip: Antidepressant Precautions – Drugs.com MedNews
April 19, 2019 - Bigger portions lead to preschoolers eating more over time
April 19, 2019 - Specific strains of Staphylococcus aureus linked to wounds that do not heal
Research establishes use of chimeric cells as potential therapy for Duchenne muscular dystrophy

Research establishes use of chimeric cells as potential therapy for Duchenne muscular dystrophy

image_pdfDownload PDFimage_print

Cells made by fusing a normal human muscle cell with a muscle cell from a person with Duchenne muscular dystrophy –a rare but fatal form of muscular dystrophy — were able to significantly improve muscle function when implanted into the muscles of a mouse model of the disease. The findings are reported by researchers from the University of Illinois at Chicago in the Stem Cell Review and Reports.

These cells, called “chimeric cells,” are made by combining a normal donor cell containing a functioning copy of the gene for dystrophin — a structural muscle protein lacking in people with Duchenne muscular dystrophy — with a cell from a recipient with the disease. In a January 2018 paper in Stem Cell Reviews and Reports, the researchers used mouse donor and recipient cells to make chimeric cells that boosted dystrophin levels by 37 percent and improved muscle function when implanted into the muscles of a mouse model of Duchenne muscular dystrophy. The new cells had both donor and recipient characteristics and interacted with their surroundings like normal cells. The chimeric cells remained viable and produced dystrophin for 30 days.

Now, the research group, led by Dr. Maria Siemionow, professor of orthopedic surgery in the UIC College of Medicine, report similar findings using human cells implanted in a mouse model of Duchenne muscular dystrophy.

“Our results point to the long-term survival of these cells and helps establish the use of chimeric cells as a novel promising potential therapy for patients with Duchenne muscular dystrophy,” Siemionow said. Her team is looking forward to clinical trials in humans in the near future, she said.

Duchenne muscular dystrophy is an X-linked genetic disorder characterized by progressive muscle degeneration and weakness, affecting approximately 1 in 4,000 newborn boys.

People with the disease don’t have the gene for dystrophin, a structural protein that helps keep muscle cells intact. Symptom onset is in early childhood, usually between ages 3 and 5. The disorder causes muscle weakness and loss of motor function and ultimately results in respiratory or cardiac failure and death. With advances in treatment, many with Duchenne muscular dystrophy live into their teens and 20s, and some into their 30s, but there is currently no cure for the disease.

Promising treatments include gene therapy and stem cell therapy, but each has drawbacks. Gene therapy relies on delivering good copies of missing or dysfunctional genes to cells via viruses. Not only can cells become immune to viral infection, rendering the therapy ineffective, but there also is no guarantee that viruses will only infect the intended cells. Stem cell therapy, where cells that contain the dystrophin gene are implanted into a recipient, requires that the recipient take immunosuppressive drugs to prevent rejection.

“This is not conventional stem cell therapy,” said Siemionow, who is the lead author of the paper. “We are restoring dystrophin in such a way that the recipient won’t need to take anti-rejection therapy because the implanted chimeric cells can evade the recipient’s immune system. In traditional stem cell therapy, the implanted cells are 100 percent ‘other’ and anti-rejection medicine is needed in order to prevent the host immune system from destroying those foreign cells.”

In contrast, chimeric cells are 50 percent “self” — with many biochemical and genetic features of the recipient — and as such can trick the recipient’s immune system into ignoring them.

“The chimeric cells are just enough like the recipient’s own cells that their immune system gives them a ‘pass’ so to speak,” Siemionow said.

If such cells were to be used to treat a patient with Duchenne muscular dystrophy, then normal muscle cells from the recipient’s father or close relative would be fused with muscle cells from the patient.

The researchers fused muscle cells from patients with Duchenne muscular dystrophy with muscle cells from normal, healthy donors.

In the lab, the chimeric cells were seen to express dystrophin.

When the cells were implanted into the leg muscles of a mouse model of Duchenne muscular dystrophy, dystrophin levels rose approximately 20 percent of muscle fibers affected by the implanted cells at 90 days post-implantation, “enough to produce a significant improvement in muscle function,” said Dr. Kris Siemionow, associate professor of orthopedic surgery in the UIC College of Medicine and a co-author on the paper.

Improvements of more than 60 percent were seen in muscle function tests of the implanted mice, and improvements of more than 20 percent in tests of muscle fatigue tolerance.

Maria and Kris Siemionow, who are mother and son, have recently launched a company to develop their chimeric cells into a therapy to treat Duchenne muscular dystrophy called Dystrogen Therapeutics.

Tagged with:

About author

Related Articles