Novel gene therapy offers potential treatment for FCMD
Japanese researchers proposed using RNA interference for correcting a mistake in the genetic code of patients with Fukuyama Muscular Dystrophy (FCMD).
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Misfolding proteins bring caution for gene therapies for haemophilia
US researchers have discovered a link between protein misfolding and liver cancer, that could help improve gene therapy for haemophilia.
New machine learning model could encode words to kill cancer cells
US researchers, using new machine learning techniques have developed a virtual molecular library of “words” that encode commands to kill cancer cells.
New ALSV virus discovered in Swiss ticks
Researchers have detected Alongshan virus (ALSV) for the first time in Switzerland in ticks.
P53 could be key to therapies for salivary gland cancer
Mouse models show that activating a non-mutated form of the gene could lead to developing therapies for salivary gland cancer.
CAR-T therapy becomes a powerful tool against an autoimmune disease like MS
US researchers have shown that CAR-T therapy can be used to eliminate unwanted cells that cause autoimmunity.
Where should the priority be for monkeypox vaccine allocations?
Canadian researchers underwent a modelling study to explore optimal allocation of vaccines against monkeypox virus (MPXV), that prioritises vaccines to larger networks with more initial infections and greater potential for spread is best.
CIRM grant $4.8m to fund gene therapy for rare disease
CIRM grant will fund novel gene therapy that aims for single lifelong treatment of Friedreich’s ataxia, a progressive neuromuscular disorder; a second CIRM grant will advance efforts to leverage UC San Diego research on another rare disease
New target in the fight against heart disease
US scientists have new insight into how heart cells enable unhealthy growth, and identify a new target to intervene against heart disease.
Researchers have engineered AAV vectors that cross the blood-brain barrier
In primate models, researchers have successfully shown that developed AAVs can cross the blood-brain barrier, which keeps many drugs from getting into the brain.
Immune cells in ALS patients: predicting disease progression
Swedish researchers have discovered that by measuring immune cells in the cerebrospinal fluid when diagnosing ALS, it is possible to predict how fast the disease may progress.
What parts of a tumour are linked to muscle wasting?
US researchers found that the severity of muscle wasting from cancer is related to the type, size and location of the tumour, in mice.
Deteriorating neurons: the source of human brain inflammation in Alzheimer’s disease
US researchers administered a therapy to Alzheimer’s patient-derived neurons in the lab, eliminating deteriorating cells, leading to positive consequences for the remaining healthy cells.
Snapshots of a mouse brain show OPCs nibbling on neurons
A US researcher studied 3D structures of brain cells and connections, revealing new role for oligodendrocyte precursor cells (OPCs), in mice.
Novel nanoparticles deliver innovative cancer chemoimmunotherapy
University of Pittsburgh researchers have designed cancer-fighting nanoparticles that co-deliver a chemotherapy drug and a novel immunotherapy