Breaking News
July 19, 2018 - Refining standards of maternal-fetal care
July 19, 2018 - Stitching single cells together any which way you want to
July 19, 2018 - Study identifies RNA molecules that regulate male hormones in prostate cancer
July 19, 2018 - New machine-learning model shows promise in predicting undiagnosed dementia
July 19, 2018 - Sleep supports antioxidant processes, study suggests
July 19, 2018 - MiRagen Therapeutics Announces Initiation of Phase 2 Clinical Trial of MRG-201
July 19, 2018 - Unique brain ‘fingerprint’ can predict drug effectiveness
July 19, 2018 - Life on the border: Struggling to survive in Jordan
July 19, 2018 - CT scans may raise brain tumor risk
July 19, 2018 - Moderate alcohol intake linked with improved male fertility
July 19, 2018 - Alcohol-related cirrhosis mortality on the rise among young adults
July 19, 2018 - Study uncovers new protein complex that shields broken DNA ends
July 19, 2018 - Regular sunscreen use protects young people from melanoma
July 19, 2018 - Using non-invasive brain recordings to characterize activity in deep structures
July 19, 2018 - Mediterranean diet could influence academic performance through effects on sleep quality
July 19, 2018 - Woman’s pregnancy history may be linked to Alzheimer’s disease risk
July 19, 2018 - Study calls for new gold standard in research to treat non-alcoholic steatohepatitis
July 19, 2018 - Cancer patients receiving anti-PD-1 therapies may experience delayed skin reactions
July 19, 2018 - Scientists study adverse effects of carbon, silicon nanotubes and carbon nanofibers
July 19, 2018 - Keck Hospital of USC receives Magnet recognition for excellence in nursing
July 19, 2018 - Scientists identify hidden signals in RNAs that control protein synthesis
July 19, 2018 - Quadrivalent HPV Vaccine Not Tied to Spontaneous Abortion
July 19, 2018 - FDA OKs first drug made to reduce excessive sweating
July 19, 2018 - New findings do no support caffeine as effective appetite suppressant or weight-loss aid
July 19, 2018 - Study evaluates Neoteryx’s VAMS technology for monitoring HbA1c levels of diabetic children
July 19, 2018 - New clinical trial examines use of adrenaline to treat cardiac arrests
July 19, 2018 - Early surgical intervention may improve outcomes for patients with mitral valve disease
July 19, 2018 - Prolonged preoperative opioid use linked to adverse outcomes after total knee and hip arthroplasty
July 19, 2018 - Biophysicists use infrared sensor as new method for drug discovery
July 19, 2018 - Rat study shows negative effects of perinatal exposure to phthalates
July 19, 2018 - Children with disabilities endure long waits for life-changing medical equipment
July 19, 2018 - Ways to stay safe while camping and hiking
July 19, 2018 - People with HIV twice as likely to suffer from heart disease
July 19, 2018 - On-the-Job Stress Relief – Drugs.com MedNews
July 19, 2018 - Compounds found in green tea and wine may block formation of toxic metabolites
July 19, 2018 - Gene regulator associated with protein pileup in exfoliation glaucoma
July 19, 2018 - Trump administration summons immigrant infants
July 19, 2018 - FDA grants approval for first breast cancer drug through ‘Real-Time Oncology Review’
July 19, 2018 - Five tips for men seeking plastic surgery
July 19, 2018 - Researchers discover the reasons why some people get dizzy when hearing certain sounds
July 19, 2018 - Research project investigates snake venom treatment as antibiotic alternative for eye infections
July 19, 2018 - Melanoma could soon be detected using a blood test
July 19, 2018 - Exposure to bright light may have big impact on sleep-related behavior in children
July 19, 2018 - Deleting single gene in gut bacteria affects metabolism, reduces weight gain in mice
July 19, 2018 - New proteomics studies help gain more insights into Alzheimer’s, cancer and listeriosis
July 19, 2018 - Study finds major discrepancies in prescription drug labeling pregnancy information across four countries
July 19, 2018 - Cellectar’s CLR 131 Receives FDA Orphan Drug Designation for Treatment of Ewing’s Sarcoma
July 19, 2018 - Watching the immune system in action reveals what happens when things goes wrong
July 19, 2018 - Increasing blood sugar levels improves memory and performance in older adults
July 19, 2018 - Connection between self-regulation and obesity appears to be different for girls and boys
July 19, 2018 - Researchers develop new, less destructive method for whitening teeth
July 19, 2018 - Revving up innate control of viral infection requires a three-cell ignition
July 19, 2018 - Inaccurate direct-to-consumer raw genetic data can harm patients, new research suggests
July 19, 2018 - Weight loss surgery is effective under the right situations
July 19, 2018 - BioTek awarded patent for autofocus feature on microplate reader
July 19, 2018 - Low-carb diets reduce stiffness of arteries in women and promote weight loss in men
July 19, 2018 - New review examines cannabinoids’ potential for direct treatment of cancer
July 19, 2018 - Allergic responses may help protect the skin against cancer, research suggests
July 19, 2018 - Inappropriate Prescribing of Abx High in Urgent Care Centers
July 19, 2018 - Many at risk for HIV despite lifesaving pill
July 19, 2018 - Tips for doctors and parents on the harms of marijuana use for teens
July 18, 2018 - Researchers detect presence of IgE antibodies after kidney transplantation
July 18, 2018 - New technique allows researchers to create large scale, personalized bone grafts
July 18, 2018 - Smoking May Boost Atrial Fibrillation Risk
July 18, 2018 - Genome editing method targets AIDS virus
July 18, 2018 - These things matter: Medical complications are not inevitable, a physician writes
July 18, 2018 - Cognitive functions often wilt as water departs the body, shows study
July 18, 2018 - Origins of bread found 14,400 years ago in Jordan
July 18, 2018 - Low-dose ketamine found to be as effective as opioids for treating acute pain
July 18, 2018 - Novel bioengineering technique could help repair bone defects
July 18, 2018 - Researchers identify new potential target protein for colon cancer
July 18, 2018 - Air pollution contributes significantly to diabetes globally
July 18, 2018 - Cell membrane’s importance offers new strategy to fight infections
July 18, 2018 - Researchers identify key protein involved in irregular brain cell activity
July 18, 2018 - 3D modeling of drug resistance could lead to more effective cancer treatment
July 18, 2018 - Hunger hormones could be key to new treatments for drug, alcohol addiction
July 18, 2018 - Nitrate-cured meats may contribute to mania, study finds
July 18, 2018 - Why men may recover more quickly from influenza infections than women
July 18, 2018 - Study finds discharge against medical advice as predictor of readmissions in heart attack patients
July 18, 2018 - KemPharm Announces Top Line Results from KP415.E01 Efficacy and Safety Trial in Children With ADHD
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.

Source:

Human ‘chimeric’ cells restore crucial protein in Duchenne muscular dystrophy

Tagged with:

About author

Related Articles