New research has identified a potential method for treating fragile X syndrome, a leading cause of autism spectrum disorders that is characterized by an inherited repeat of certain nucleotides within the DNA sequence of the ...
May 19, 2023
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115
In a major new development in the quest to develop better gene therapies with which to treat a host of diseases, researchers at the University of Massachusetts Amherst and UMass Chan Medical School recently announced that ...
Jul 25, 2022
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42
A new study led by scientists at VCU Massey Cancer Center has shown that an experimental drug known as AZ32 selectively sensitizes brain tumors to radiation and significantly extends the survival of mouse models with human ...
Jun 27, 2018
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253
A recently developed system for switching on the activity of genes could improve treatments for a broad range of neurological diseases. Esteban Engel, a researcher in viral neuroengineering in the Princeton Neuroscience Institute, ...
Apr 17, 2020
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344
A Northwestern Medicine study has identified a molecular mechanism that helps regulate the progression of castration-resistant prostate cancer, according to findings published in the journal Oncogene.
Sep 5, 2022
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995
By 2050, one in 10 individuals are expected to live with some form of hearing loss. Of the hundreds of millions of cases of hearing loss affecting individuals worldwide, genetic hearing loss is often the most difficult to ...
May 26, 2023
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Two discoveries at UC Santa Barbara point to potential new drug therapies for patients with kidney disease. The findings are published in this week's issue of the Proceedings of the National Academy of Sciences.
Apr 26, 2011
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A promising therapy for a range of brain diseases involves antisense oligonucleotides (ASOs)—specialized molecules that can modulate RNA and alter protein production—directly injected into the cerebrospinal fluid, in ...
Mar 13, 2023
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575
Creating "super soldiers" of specific white blood cells to boost an anti-tumor response has been shown in a series of elegant experiments by Princess Margaret researchers.
Feb 19, 2021
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230
UCLA researchers led by Dr. Donald Kohn have created a method for modifying blood stem cells to reverse the genetic mutation that causes a life-threatening autoimmune syndrome called IPEX. The gene therapy, which was tested ...
Jan 11, 2019
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400
