Breaking News
April 27, 2018 - What happens to our muscles during spaceflight and when living on Mars?
April 27, 2018 - Abdominal pain should be seen as a warning sign for invasive meningococcal disease
April 26, 2018 - Mediterranean diet enhances good bacteria living in the gut
April 26, 2018 - UA researchers aim to predict and prevent fatal disease in premature babies
April 26, 2018 - Researchers develop new method for early and accurate breast cancer screening
April 26, 2018 - Cytochalasin B-induced membrane vesicles successfully tested as vector for anti-tumor drug delivery
April 26, 2018 - Hospitals lure diabetes patients with self-care courses, but costs can weigh heavily
April 26, 2018 - Common therapy options for psoriasis can help reduce coronary plaque, study shows
April 26, 2018 - Kids Are Naturally as Fit as an ‘Iron Man’
April 26, 2018 - Traditional health claims about India’s ayurvedic foods help make them big business
April 26, 2018 - Maternal binge drinking may make offspring more vulnerable to mood problems and alcohol abuse
April 26, 2018 - VA hospitals provide better quality care than other health providers
April 26, 2018 - Cytosurge develops new 3D printing method that allows scientists to manufacture tiny, complex metal components
April 26, 2018 - Detailed genetic testing of head and neck cancers could lead to more personalized treatments
April 26, 2018 - Study shows new approach to improve survival rates for patients with stage IV Wilms tumors
April 26, 2018 - Nonsurgical technique enables people with spinal cord injuries to regain use of hands
April 26, 2018 - Elective induction of labor at 39 weeks decreases risks of C-section, other complications
April 26, 2018 - Hair products used by Black women contain dozens of hazardous chemicals
April 26, 2018 - Scientists create an atlas of the human genome
April 26, 2018 - New shape of DNA found in human cells
April 26, 2018 - Researchers target telomerase for killing NRAS-mutant melanoma cells
April 26, 2018 - Scientists improve growth of 3d brain models to better understand autism, dementia, schizophrenia
April 26, 2018 - Evox Therapeutics expands into new facilities at The Oxford Science Park
April 26, 2018 - Scientists discover new method for measuring cellular age
April 26, 2018 - UNC-Chapel Hill receives new award to help improve health of North Carolinians
April 26, 2018 - Study finds no evidence of chronic wasting disease transmissibility in macaques
April 26, 2018 - Study highlights impact of early detection on skin cancer survival
April 26, 2018 - Scientists discover culprit in reducing effectiveness of insulin
April 26, 2018 - Exploiting rabies virus machinery to usher Parkinson’s disease drug directly to the brain
April 26, 2018 - ‘Rapid autopsy’ programs seek clues to cancer within hours of death
April 26, 2018 - Personalised prescriptions according to your genetics
April 26, 2018 - ECDC analysis highlights measles vaccination gaps in teenagers and young adults
April 26, 2018 - Study assesses link between cognitive function levels and crash risk among older drivers
April 26, 2018 - Researchers uncover how obesity leads to cancer initiation
April 26, 2018 - Consuming large quantities of sugar during pregnancy can affect child’s cognition, shows study
April 26, 2018 - Researchers create new tool to measure patient uncertainty for predicting hospital readmissions
April 26, 2018 - FDA Alert: Lamictal (lamotrigine): Drug Safety Communication
April 26, 2018 - Massive single-cell survey of kidney cell types reveals new paths to disease
April 26, 2018 - Cognitive behavioral therapy can help children with autism manage emotional challenges
April 26, 2018 - CU Anschutz Medical Campus receives NIH grant to speed up discovery of new treatments
April 26, 2018 - Researchers discover significant distortions in leading genetics study method
April 26, 2018 - New combination therapy could improve survival in children with high-risk neuroblastoma
April 26, 2018 - Scientists clarify casual role of oxidative stress in metabolically abnormal and healthy obesities
April 26, 2018 - Home-based exercise program found ineffective for patients with peripheral artery disease
April 26, 2018 - Surgeries performed by older surgeons have lower patient mortality rates, study reveals
April 26, 2018 - New UCLA study could elucidate certain causes of infertility and miscarriage
April 26, 2018 - Choroidal Thickness Changes in Patients With Untreated DM
April 26, 2018 - Medical chemists discover peptic ulcer treatment metallodrug effective in ‘taming’ superbugs
April 26, 2018 - UB researchers build 3D-printed, drug-filled dentures to fight against infections
April 26, 2018 - Researchers find role of iron storage gene in slowing down prostate cancer growth
April 26, 2018 - Study suggests link between regular mid-day naps and neurocognitive function in teens
April 26, 2018 - Researchers gain ground-breaking insights into how inflammatory diseases work
April 26, 2018 - Injured U.S. Vet Receives World’s First Penis/Scrotum Transplant
April 26, 2018 - Researchers find existing drug effective at preventing onset of type 1 diabetes
April 26, 2018 - MGH researchers identify risk factors for drug overdose in youth with substance use disorders
April 26, 2018 - Researchers develop new vaccine to help people overcome bath salts abuse
April 26, 2018 - Genetic signature predicts diabetes diagnosis
April 26, 2018 - Study shows link between restless legs syndrome symptoms and brain structure
April 26, 2018 - MU researchers use new techniques to fight against diabetic retinopathy
April 26, 2018 - AAE’s new practice statements aim at improving patient care
April 26, 2018 - Expression of long non-coding RNAs can result in high levels of specific proteins involved in cancer
April 26, 2018 - FDA re-examining safety of new drug approved for Parkinson’s disease psychosis
April 26, 2018 - Unanimous Positive Result of FDA Advisory Committee Meeting for First Plant-Based Pharmaceutical Cannabidiol Treatment for Seizures in Patients with Two Rare, Severe Forms of Epilepsy
April 26, 2018 - Bacteria boost antifungal drug resistance in severe childhood tooth decay
April 26, 2018 - New study affirms bedtime habits of Americans
April 26, 2018 - Hospital patients are more interested in tracking their health data, research shows
April 26, 2018 - Study shows gene variations associated with malaria risk
April 26, 2018 - Inhealthcare teams up with MSKassist to combat problems related to obesity and aging
April 26, 2018 - Caffeine during pregnancy – babies 66 percent more likely to become overweight
April 26, 2018 - FDA Approves Jynarque (tolvaptan) to Slow Kidney Function Decline in Rapidly Progressing Autosomal Dominant Polycystic Kidney Disease
April 26, 2018 - Pricey dental implants often best but insurance rarely pays
April 26, 2018 - Advion’s Peak Express software now available with the expression compact mass spectrometer
April 25, 2018 - Researchers find link between pneumonia in older people and PPI prescriptions
April 25, 2018 - Alcohol damages microbiome in the mouth
April 25, 2018 - Regular soaking in hot tub improves health outlook in obese women with PCOS
April 25, 2018 - FDA Alert: Magnetic Resonance-guided Laser Interstitial Thermal Therapy (MRgLITT) Devices: Letter to Health Care Providers
April 25, 2018 - Sunshine could hold clues on the timing for a severe form of heart attack, study says
April 25, 2018 - Sartorius Stedim Biotech launches mini bioreactor vessel for ambr 250 high throughput system
April 25, 2018 - Biofeedback-assisted relaxation may help children during medical procedures
April 25, 2018 - Key signaling protein in Huntington’s disease found to have deleterious effects on heart function, shows study
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