Medical research

Using gene therapy to tackle complex brain disease

Substantial progress has been made in the development of treatments for a particular brain disease, thanks in part to an EU-funded project. The X-ALD project focused on achieving a better understanding of the pathogenic mechanisms ...

Medical research

Unraveling the mechanism of antisense oligonucleotides

Stanley T. Crooke, MD, PhD, CEO of Ionis Pharmaceuticals and recipient of the 2016 Lifetime Achievement Award from the Oligonucleotide Therapeutic Society presents a detailed look at the mechanisms that underlie antisense ...

Genetics

Research team helps father discover source of son's disability

The severe learning disabilities of a teenager in the Netherlands were caused by a genetic mutation that affects the function of ribosomes, the cellular protein-making factories crucial to all life. The new finding by an ...

Genetics

Candidate drug provides benefit in SMA animal models

In a new publication that appears in Human Molecular Genetics, the laboratory of Christine DiDonato, PhD reports on their pharmacological characterization of the drug RG3039, demonstrating that it can extend survival and ...

Oncology & Cancer

Laying siege to chemoresistance

To date, tests have only been carried out on cells, but a piece of research conducted by the Department of Genetics at the UPV/EHU's Faculty of Medicine in collaboration with MD Anderson and the CNIO is opening up the door ...

Medications

New guide highlights the properties of diverse drug targets

The new Concise Guide to PHARMACOLOGY 2015/2016 provides a valuable and unique overview of the key properties of more than 1,700 human drug targets, focusing on those exploited currently in the clinic or with future therapeutic ...

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