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New UCI-led study lays foundation for establishing base editing as a one-time, durable treatment for inherited retinal degeneration

A new UCI-led study indicates base editing has tremendous potential to treat inherited retinal degeneration, particularly in Leber congenital amaurosis (LCA) patients. The treatment rescued cone photoreceptors on a long-term basis, suggesting a one-time, durable treatment for LCA patients.

SourceUniversity of California - Irvine·JournalNature Communications·DateApr 5, 2022

An approach to treating a severe congenital myopathy

A new treatment approach has been proposed for children with congenital muscle disorders, using inhibitors to improve muscle function and movement in mice with mutations in the RYR1 gene. The therapy targets genetic material overload caused by excessive enzyme production, leading to reduced access of genes to cellular machinery.

SourceUniversity of Basel·JournaleLife·TypeExperimental study·DateMar 29, 2022

Steven A. Rosenberg, MD, PhD, FAACR, recognized with 2022 Pezcoller Foundation-AACR International Award for Extraordinary Achievement in Cancer Research

Dr. Steven A. Rosenberg's pioneering work on IL-2 and its use in treating metastatic melanoma and other cancers led to the FDA approval of first U.S.-born cancer immunotherapy. His subsequent research on CAR T-cell therapy has resulted in promising clinical results for various types of cancer.

Tufts CTSI publishes first-of-its-kind resource on real-world research conducted with broadly engaged, diverse, and inclusive teams

The Tufts Clinical and Translational Science Institute has published a comprehensive resource on broadly engaged team science, which aims to include stakeholders in the research process. The book features perspectives and best practices from over 60 experts to inspire researchers and stakeholders to build diverse, inclusive teams.

Molecular pathway driving maladaptive processes in heart failure elucidated by scientists at Lewis Katz School of Medicine at Temple University, opening door to new therapeutic strategies

Researchers at Lewis Katz School of Medicine at Temple University discover a molecular pathway driving maladaptive processes in heart failure, revealing the importance of GRK5's catalytic activity and nuclear presence. The study opens doors to new therapeutic strategies for treating this devastating condition.

SourceTemple University Health System·JournalJournal of the American College of Cardiology·DateMar 15, 2022

The Green Mediterranean diet modifies the bacterial population in our gut, with specific bacteria mediating weight loss and cardiac risk reduction

The green Mediterranean diet induces specific microbial changes in the gut microbiome, enriching with bacteria that positively affect glucose metabolism and insulin sensitivity. This novel diet also promotes genetic pathways involved in reducing branched-chain amino acids linked to insulin resistance.

SourceBen-Gurion University of the Negev·JournalGenome Medicine·TypeRandomized controlled/clinical trial·DateMar 10, 2022

O-pH, a new UW dental tool prototype, can spot the acidic conditions that lead to cavities

Researchers created a dental tool that measures plaque acidity using an LED light and FDA-approved chemical dye, providing dentists with early warning signs of cavity development. The device can help limit the need for specific harmful bacteria tests and educate patients about sugar's impact on oral health.

SourceUniversity of Washington·JournalIEEE Transactions on Biomedical Engineering·TypeExperimental study·DateMar 8, 2022

Researchers from the GIST propose ultrasound stimulation as an effective therapy for Alzheimer's disease in new study

Researchers at GIST demonstrate that ultrasound pulses can reduce β-amyloid plaque concentration and tau protein levels in AD-model mice, suggesting a promising non-invasive therapeutic strategy. The treatment also improves brain connectivity without causing harm to brain tissue.

SourceGIST (Gwangju Institute of Science and Technology)·JournalTranslational Neurodegeneration·TypeExperimental study·DateJan 24, 2022

Penn Medicine researchers develop new method to increase effectiveness of nanomedicines

Researchers at Penn Medicine have discovered a new method to prevent the body's proteins from attacking treatment-carrying nanoparticles, allowing for more effective delivery of therapies. By coating nanoparticles with natural suppressors of complement activation, such as Factor I, the team has shown improved protection against immune ...

SourceUniversity of Pennsylvania School of Medicine·JournalAdvanced Materials·TypeExperimental study·DateJan 11, 2022

Experimental treatment with enzyme protects mice from lethal anthrax infection

Scientists have developed an enzyme that degrades the capsule surrounding the bacterium that causes anthrax, reducing virulence and protecting mice from infection. The treatment, known as PEG-CapD-CPS334C, is a promising avenue for treating multidrug-resistant anthrax and other bacterial infections.

SourceUS Army Medical Research Institute of Infectious Diseases·JournalScience Translational Medicine·TypeExperimental study·DateDec 8, 2021

Researchers overcome translational gap by developing novel in vivo model for the labeling of insulin secretory granule pools

A transgenic pig model has been developed allowing for the first time the in vivo fluorescent labeling of age-distinct insulin secretory granule pools. This model enables researchers to study insulin turnover in normoglycemic conditions, closing the translational gap between humans and rodents.

SourceDeutsches Zentrum fuer Diabetesforschung DZD·JournalProceedings of the National Academy of Sciences·TypeExperimental study·DateSep 29, 2021

Researchers report on new way to deliver healing stem cells to kidney injury patients

A new ex vivo drug delivery system has been shown to keep MSCs viable longer and reprogram the peripheral immune response toward organ repair. The study's results suggest that SBI-101 can impact a peripheral immune response that can be transmitted to local tissue damage in vital organs, including the kidney.

SourceAlphaMed Press·JournalStem Cells Translational Medicine·TypeRandomized controlled/clinical trial·DateSep 28, 2021

‘Research autopsy’ helps scientists study why certain cancer therapies stop working

A new study at Ohio State University's Comprehensive Cancer Center is using rapid autopsies to gather biological samples after death to better understand how cancer cells overcome different treatments. This approach has already led to novel findings about drug resistance mechanisms, including the recent approval of a targeted therapy f...

SourceOhio State University Wexner Medical Center·JournalThe Lancet Gastroenterology & Hepatology·TypeExperimental study·DateSep 28, 2021

A 3D printed vaccine patch offers vaccination without a shot

Scientists at Stanford and UNC Chapel Hill developed a 3D-printed vaccine patch that provides greater protection than traditional vaccines. The patch applies the vaccine directly to the skin, triggering a potent immune response and potentially leading to dose sparing and higher vaccination rates.

SourceUniversity of North Carolina at Chapel Hill·JournalProceedings of the National Academy of Sciences·TypeExperimental study·DateSep 24, 2021

Repeated injury to airway stem cells could be major factor in chronic lung disease

Researchers link repeated injury to airway stem cells with chronic lung disease, suggesting that biological aging of these cells may contribute to the development of this condition. The study found that injury caused activation of a subset of stem cells, leading to their premature aging and loss of functional capacity.

SourceAlphaMed Press·JournalStem Cells Translational Medicine·TypeExperimental study·DateSep 21, 2021

Phase 1 clinical study reveals how stem cells alleviate COPD inflammation in humans

A new clinical study reveals that treatment with mesenchymal stromal cells reduces inflammation in patients with chronic obstructive pulmonary disease (COPD). The study provides insights into the therapeutic potential of MSCs as a novel treatment for COPD, which is a leading cause of morbidity and mortality worldwide.

SourceAlphaMed Press·JournalStem Cells Translational Medicine·TypeRandomized controlled/clinical trial·DateAug 18, 2021

People given 'friendly' bacteria in nose drops protected against meningitis

Researchers have successfully used nose drops containing genetically modified friendly bacteria to protect against meningitis, showing strong and long-lasting immune responses. The new method, which exploits the natural presence of non-pathogenic Neisseria lactamica in the nose, has potential applications beyond meningitis prevention.

SourceUniversity of Southampton·JournalScience Translational Medicine·DateJul 12, 2021

Immune cells in the human biliary system mapped

The study reveals a dominant network of neutrophils and tissue-resident T cells in the bile ducts of patients with primary sclerosing cholangitis (PSC), a severe inflammatory disease. This discovery may pave the way for new treatment strategies against PSC and other biliary disorders.

SourceKarolinska Institutet·JournalScience Translational Medicine·DateJun 23, 2021

Speeding new treatments

Researchers at the University of New Mexico Health Sciences Center have created a powerful computational tool called REDIAL-2020. This online suite of models enables scientists to quickly identify small molecules capable of disarming COVID-19, narrowing the field for future experiments and accelerating the discovery process.

SourceUniversity of New Mexico Health Sciences Center·JournalNature Machine Intelligence·DateMay 3, 2021