Gene Therapy-Associated Uveitis (GTAU): Understanding and mitigating the adverse immune response in retinal gene therapy.

Retinal gene therapy using adeno-associated viral (AAV) vectors has been a groundbreaking step-change in the treatment of inherited retinal diseases (IRDs) and could also be used to treat more common retinal diseases such as age-related macular degeneration and diabetic retinopathy. The delivery and expression of therapeutic transgenes in the eye is limited by innate and adaptive immune responses against components of the vector product, which has been termed gene therapy-associated uveitis (GTAU). This is clinically important as intraocular inflammation could lead to irreversible loss of retinal cells, deterioration of visual function and reduced durability of treatment effect associated with a costly one-off treatment.

Vacancies-rich Z-scheme VdW heterojunction as HS-sensitized synergistic therapeutic nanoplatform against refractory biofilm infections.

Encapsulated in a self-produced negatively charged extracellular polymeric substance (EPS) matrix, the wound infected bacterial biofilms exhibit formidable resistance to conventional positively charged antibiotics and host's immune responses, which can undoubtedly lead to persistent infections and lethal complications. Nevertheless, developing efficacious strategies to root out stubborn biofilm and promote tissue regeneration still remains a challenge. To resolve this dilemma, a versatile vacancies-rich Z-scheme MoSSe Van der Waals heterojunction (MoSSe VdW HJ) is rationally fabricated as nanoplatform for hydrogen sulfide (HS)-sensitized synergistic therapy of wound bacterial biofilm infection.

Bladder outlet procedures without augmentation cystoplasty: Long-term outcomes and association of preoperative bladder compliance with subsequent augmentation cystoplasty or urinary diversion and DMSA abnormalities.

Background: A bladder outlet procedure (BOP) without augmentation cystoplasty (AC) for incontinence from neurogenic bladder has risks including renal damage and future surgeries.

Objective: Our objective was to obtain long-term outcomes after BOP without AC and identify risk factors for adverse outcomes. We hypothesized that high preoperative bladder compliance is associated with lower risk of subsequent AC or diversion (AC/D) and dimercaptosuccinic acid renal scan (DMSA) abnormalities.

Ambulatory status and sexual function and activity in young adults with spina bifida.

Introduction: Young adults with spina bifida may experience altered genital sensation and sexual function. There are no validated questionnaires and few studies examining sexual function and activity in this population. Ambulatory status has previously been associated with better erectile function, but few studies report on other domains of sexual function in men and women.

A systematic benchmark of Nanopore long-read RNA sequencing for transcript-level analysis in human cell lines.

The human genome contains instructions to transcribe more than 200,000 RNAs. However, many RNA transcripts are generated from the same gene, resulting in alternative isoforms that are highly similar and that remain difficult to quantify. To evaluate the ability to study RNA transcript expression, we profiled seven human cell lines with five different RNA-sequencing protocols, including short-read cDNA, Nanopore long-read direct RNA, amplification-free direct cDNA and PCR-amplified cDNA sequencing, and PacBio IsoSeq, with multiple spike-in controls, and additional transcriptome-wide N-methyladenosine profiling data.