Stepped Care for Patients to Optimize Whole Recovery (SC-POWR): An Effectiveness Trial Evaluating a Stepped Care Model for Individuals With Opioid Use Disorder and Chronic Pain.

Many patients who receive treatment for opioid use disorder (OUD) report experiencing chronic pain (CP), which is associated with high levels of ongoing nonmedical opioid use and low retention in OUD treatment. In pilot studies of patients with OUD receiving buprenorphine or methadone who had CP, cognitive behavioral therapy (CBT) attenuated nonmedical opioid use compared with treatment-as-usual (TAU), but patients in both treatment arms exhibited similar pain improvements. Adding exercise and stress reduction to this model may augment pain-related outcomes.

RNA Interference Effectors Selectively Silence the Pathogenic Variant c.607 G > A .

RNA interference (RNAi)-based therapeutics hold the potential for dominant genetic disorders, enabling sequence-specific inhibition of pathogenic gene products. We aimed to direct RNAi for the selective suppression of the heterozygous c.607 G > A variant causing encephalopathy.

CRISPR-Cas9 correction in the DMD mouse model is accompanied by upregulation of Dp71f protein.

Duchenne muscular dystrophy (DMD) is a severe hereditary disease caused by a deficiency in the dystrophin protein. The most frequent types of disease-causing mutations in the DMD gene are frameshift deletions of one or more exons. Precision genome editing systems such as CRISPR-Cas9 have shown potential to restore open reading frames in numerous animal studies.

In-Frame Deletion of Dystrophin Exons 8-50 Results in DMD Phenotype.

Mutations that prevent the production of proteins in the gene cause Duchenne muscular dystrophy. Most frequently, these are deletions leading to reading-frame shift. The "reading-frame rule" states that deletions that preserve ORF result in a milder Becker muscular dystrophy.

CRISPR/Cas9-generated mouse model with humanizing single-base substitution in the for safety studies of RNA therapeutics.

The development of personalized medicine for genetic diseases requires preclinical testing in the appropriate animal models. GNAO1 encephalopathy is a severe neurodevelopmental disorder caused by heterozygous mutations in the gene. c.