Effects of one-to-one music therapy in older adults with cognitive impairment: A randomized controlled trial.

This study investigated the effects of music therapy (MT), a nonpharmacological therapy, on cognitive, behavioral, and physiological outcomes in older adults with mild-to-moderate cognitive decline residing in care home settings. A randomized controlled trial design was employed, with 42 care home residents (Mage = 86.25 years) randomly assigned to either a one-to-one 16-week MT intervention or an active control group receiving storytelling.

Systemic in utero gene editing as a treatment for cystic fibrosis.

In utero gene editing has the potential to modify disease causing genes in multiple developing tissues before birth, possibly allowing for normal organ development, disease improvement, and conceivably, cure. In cystic fibrosis (CF), a disease that arises from mutations in the cystic fibrosis transmembrane conductance regulator ( ) gene, there are signs of multiorgan disease affecting the function of the respiratory, gastrointestinal, and reproductive systems already present at birth. Thus, treating CF patients early is crucial for preventing or delaying irreversible organ damage.

Next generation triplex-forming PNAs for site-specific genome editing of the F508del CFTR mutation.

Background: Cystic Fibrosis (CF) is an autosomal recessive genetic disease caused by mutations in the gene encoding the cystic fibrosis transmembrane conductance regulator (CFTR) protein for which there is no cure. One approach to cure CF is to correct the underlying mutations in the CFTR gene. We have used triplex-forming peptide nucleic acids (PNAs) loaded into biodegradable nanoparticles (NPs) in combination with donor DNAs as reagents for correcting mutations associated with genetic diseases including CF.

Prediction of and Variants with Conflicting Clinical Interpretation in a Cohort of Breast Cancer Patients.

Germline alteration has been linked to an increased risk of hereditary breast and ovarian cancer syndromes. As a result, genetic testing, based on NGS, allows us to identify a high number of variants of uncertain significance (VUS) or conflicting interpretation of pathogenicity (CIP) variants. The identification of CIP/VUS is often considered inconclusive and clinically not actionable for the patients' and unaffected carriers' management.