Apr. 4, 2023 —Researchers uncover a mechanism in a retinal transporter protein that causes blindness, could lead to targeted ...
Dec. 7, 2022 —Research has found that misfolded proteins in liver cells contribute to the development of liver cancer, shedding new light on the mysterious origins of one of the world's deadliest diseases. ...
Aug. 24, 2022 —Researchers have developed a new gene therapy approach that shows promise for treating the dry form of Age Related Macular Degeneration (AMD) -- a progressive eye disease that affects up to 10% of ...
June 6, 2022 —A new study in mice finds that a gene therapy appears to correct a rare creatine deficiency disorder that commonly results in intellectual disabilities, problems with speech, involuntary movements ...
May 10, 2022 —Scientists reported on an experimental gene-therapy-like technique to restore the normal activity of the TCCF4 gene deficient in individuals with Pitt-Hopkins ...
Apr. 22, 2022 —Pancreatic cancer has a significantly poor prognosis; therefore, the development of effective treatments is an unmet clinical need. The major drawback in this field was the lack of useful model ...
Apr. 5, 2022 —Researchers have revealed how an 'accordion effect' is critical to switching off genes, in a study that transforms the fundamentals of what we know about gene silencing. The finding expands ...
Apr. 1, 2022 —一种新的血管生成微rna药物可以是一个新的option for the treatment of ischemic cardiovascular disease, according to a new study. In the study, the researchers describe a novel nuclear acting ...
Mar. 29, 2022 —The diagnosis is rare, but devastating -- children with congenital muscle disorders often never learn to walk. Until now, there was no chance of recovery, but researchers are now presenting a ...
Feb. 28, 2022 —Researchers have developed a technology, heteroduplex oligonucleotide (HDO), that silences certain genes whose high expression levels fuel disease. Adding a specific molecular tag allowed them to ...
Feb. 16, 2022 —Sickle cell anemia is an inherited blood disorder where red blood cells become sickle/crescent shaped. It causes frequent infections, swelling in the hands and legs, pain, severe tiredness and ...
Jan. 24, 2022 —Researchers have found that induced pluripotent stem cell--derived cardiomyocytes from a patient with arrhythmogenic cardiomyopathy recapitulate the reduced contractility and impaired desmosome ...
Jan. 19, 2022 —Researchers found a previously unknown gene mutation that can cause an incurable heart condition called dilated cardiomyopathy. This gene, BAC5, is important for the movement of calcium ions in the ...
Jan. 19, 2022 —New research suggests how a newly developed gene therapy can treat Dravet syndrome, a severe form of epilepsy, and potentially prolong survival for people with the ...
Jan. 4, 2022 —Medical experts have developed a new pre-clinical gene therapy for a rare disorder, known as limb-girdle muscular dystrophy (LGMD) 2B, that addresses the primary cellular deficit associated with this ...
Dec. 13, 2021 —An experimental gene therapy for sickle cell disease restored blood cells to their normal shape and eliminated severe pain crises for years after treatment, a multicenter study has ...
Dec. 13, 2021 —Researchers have identified novel changes in gene network interactions associated with cancer that may lead to new treatment targets for chemotherapy. Their work shows that more than 90 percent of ...
Dec. 8, 2021 —Sonodynamic therapy uses ultrasound in combination with drugs to release harmful reactive oxygen species (ROS) at the site of a tumor. However, the treatment isn't very effective because cancer ...
Dec. 2, 2021 —The brain is indeed a target for treating ALS (amyotrophic lateral sclerosis), scientists have discovered. This flips a long-standing belief the disease starts in the spinal motor neurons and any ...
Oct. 19, 2021 —一种新型计算平台标识top-performing viral vectors that could deliver gene therapies to the retina with maximum efficiency and ...
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