Adeno-Associated Virus Gene Therapy Development: Early Planning and Regulatory Considerations to Advance the Platform Vector Gene Therapy Program.

Gene therapy development presents multiple challenges, and early planning is vital in the successful implementation of such programs. The Platform Vector Gene Therapy (PaVe-GT) program is a National Institutes of Health (NIH) initiative developing adeno-associated virus (AAV) gene therapies for four low-prevalence rare diseases. Utilizing the platform-based approach, the program aims to incorporate efficiencies throughout the preclinical and clinical development processes followed by public dissemination of scientific and regulatory learnings.

Assessment of frailty in patients with heart failure: A new Heart Failure Frailty Score developed by Delphi consensus.

Aims: The Heart Failure Frailty Score (HFFS) is a novel, multidimensional tool to assess frailty in patients with heart failure (HF). It has been developed to overcome limitations of existing frailty assessment tools while being practical for clinical use. The HFFS reflects the concept of frailty as a multidimensional, dynamic and potentially reversible state, which increases vulnerability to stressors and risk of poor outcomes in patients with HF.

Disentangling effects of the COVID-19 pandemic and Black Lives Matter protests on implicit anti-Black bias.

Terror Management Theory (TMT) holds that mortal threats bolster people's desire to support their worldviews, which may contribute to increased outgroup bias. In 2020, two events likely increased mortality salience and death anxiety: COVID-19 and the Black Lives Matter (BLM) protests that followed George Floyd's murder. We used Project Implicit data to investigate their impact on implicit anti-Black bias, controlling for demographic variables.

The state-of-the-art of N-of-1 therapies and the IRDiRC N-of-1 development roadmap.

In recent years, a small number of people with rare diseases caused by unique genetic variants have been treated with therapies developed specifically for them. This pioneering field of genetic N-of-1 therapies is evolving rapidly, giving hope for the individualized treatment of people living with very rare diseases. In this Review, we outline the concept of N-of-1 individualized therapies, focusing on genetic therapies, and illustrate advances and challenges in the field using cases for which therapies have been successfully developed.

Does Body Mass Index Affect the Success of Two-Stage Management of Periprosthetic Joint Infection?

Background: Obesity is associated with increased infection risk after primary total joint arthroplasty. In this retrospective cohort analysis, we sought to assess the association between body mass index (BMI) and infection recurrence after two-stage revision total joint arthroplasty for periprosthetic joint infection (PJI).

Methods: Patients were grouped by BMI (< 30, 30 to 40, and ≥ 40) as non-obese, obese, and morbidly obese, and assessed for associations and timing of PJI reinfection as well as readmissions and complications.