Patients with congenital hearing loss could benefit from a gene therapy currently in development. Although there are approaches that could reverse the process in children and young people before it becomes severe, so far, adults do not have any treatment that prevents the progressive deterioration of auditory sensory cells caused by this disease.

The industry is looking, with renewed hope, to the “promise” of messenger RNA (mRNA) therapeutics for a wide range of diseases beyond COVID-19, and not only in vaccine form but also for gene and cell therapies.

New single-step genome editing techniques that enable the insertion, inversion or deletion of long DNA sequences at specified genome positions have been demonstrated in bacteria.

New single-step genome editing techniques that enable the insertion, inversion or deletion of long DNA sequences at specified genome positions have been demonstrated in bacteria.

The adverse effects of PD-1 blockers on the CNS observed in cancer patients could occur through their effects on an enzyme that activates microglia. Pharmacological inhibition of the enzyme in mice reduced microglial activation and cognitive deficit without altering the antitumor capacity of the immunotherapy.

During the basic science morning track on the last day of this year’s Annual Congress of the European Hematology Association (EHA), the attention was focused on oncogenic transcription factors and complexes considered turning points within the acute myeloid leukemia (AML) arena.

Myeloproliferative neoplasms (MPNs) can only be cured, to date, using allogeneic stem cell transplantation which, in turn, only works for up to 20% of patients. As calreticulin (CALR) frameshift mutations are the second most common cause of MPNs, targeting this endoplasmic reticulum resident protein is one of the strategies emerging at the forefront of hematological malignancies research.

Scientists from the PsychENCODE Consortium have analyzed the brain transcriptome in a coordinated series of studies to map all the cell types, genes, epigenetic factors, and molecular pathways involved in different psychiatric disorders. After a first set of projects based on bulk analysis, the second phase of this project included 14 simultaneous publications that revealed the cellular atlas of post-traumatic stress disorder and major depressive disorder, among others.

Understanding psychiatric disorders at a cellular and molecular level could provide a different perspective to design diagnostic and therapeutic tools searching for the origin of these disorders and the alterations they cause. Fourteen simultaneous studies from the PsychENCODE Consortium have delved into the cellular atlases of human neurodevelopment, reporting the broadest view of neuropsychiatry to date.

In a paper published in the May 17, 2024, online issue of Cell, investigators from the Duke Human Vaccine Institute reported that a sequence of three immunizations in the HVTN-133 trial was sufficient for the development of heterologous or broadly neutralizing antibodies that protected against several strains of HIV.