The number of deaths caused by prion diseases reaches about 30,000 annually. Only 5 months pass from the diagnosis of seemingly healthy patients to the fatal outcome of this neurodegenerative condition, and just 1 month until quality of life is completely lost. Removing the brain protein that causes this genetic or infectious disorder could be achieved thanks to new gene-silencing techniques. At a special meeting of the American Society of Gene & Cell Therapy, in “AAV-mediated epigenetic editing for prion disease,” Sonia Vallabh presented not just the data of her research, but the impact of this disease on her family and on herself.

Durable reprogramming of human T cells may now be possible thanks to a new technique based on the CRISPRoff and CRISPRon methodology. Researchers from the Arc Institute, Gladstone Institutes, and the University of California San Francisco have stably silenced or activated genes in this type of immune cell without cutting or altering its DNA, making T cells more resistant, active, and effective against tumors.

Durable reprogramming of human T cells may now be possible thanks to a new technique based on the CRISPRoff and CRISPRon methodology. Researchers from the Arc Institute, Gladstone Institutes, and the University of California San Francisco (UCSF) have stably silenced or activated genes in this type of immune cell without cutting or altering its DNA, making T cells more resistant, active, and effective against tumors.

A preclinical study presented at the 32nd Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), held in Seville Oct. 7-10, showed a new epigenetic editing technology that enables durable gene silencing using ELXRs, short for Epigenetic Long-Term X-Repressors. With this approach, scientists at Scribe Therapeutics Inc. successfully inhibited the expression of the PCSK9 gene, a key regulator of cholesterol metabolism, in human cells, mice and nonhuman primates.

As the many challenges facing cell therapies are being addressed, the CAR T field continues to evolve beyond its original design of T cells engineered to target hematological malignancies. During the 32nd Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), held in Seville Oct. 7-10, several studies showed how this technology is being redefined as programmable and adaptable immune cells with expanded functional versatility.

Experimental drugs that directly inhibit the NSD2 enzyme have shown potential as an effective strategy against hard-to-treat cancers, such as lung and pancreatic tumors driven by KRAS mutations. The therapeutic mechanism involves reversing a histone H3 methylation that promotes open chromatin and the expression of oncogenes.

Experimental drugs that directly inhibit the NSD2 enzyme have shown potential as an effective strategy against hard-to-treat cancers, such as lung and pancreatic tumors driven by KRAS mutations. The therapeutic mechanism involves reversing a histone H3 methylation that promotes open chromatin and the expression of oncogenes.

While people living with HIV can lead virtually normal lives thanks to antiretroviral therapy (ART), HIV persists in a latent state within cellular reservoirs that scientists do not know how to eliminate. “Transcription is a critical step in the viral life cycle. … But there are currently no drugs suppressing HIV transcription, and that may be one of the reasons why current antiretroviral therapy is not curative,” Melanie Ott told the audience at the 13th IAS Conference on HIV Science this week in Kigali, Rwanda.

The variety of blood cells decreases with age. Some are lost, while others become dominant, leading to a loss of functional diversity. This, in turn, weakens the immune system in older individuals and increases the risk of developing hematological diseases. Scientists in Barcelona have developed a method based on DNA methylation that works like a barcode. EPI-Clone identifies and traces the origin of blood cells to measure the complexity of these clones in aging humans and mice.

The variety of blood cells decreases with age. Some are lost, while others become dominant, leading to a loss of functional diversity. This, in turn, weakens the immune system in older individuals and increases the risk of developing hematological diseases. Scientists in Barcelona have developed a method based on DNA methylation that works like a barcode. EPI-Clone identifies and traces the origin of blood cells to measure the complexity of these clones in aging humans and mice.