All-in-one gene therapy alternative for DBAS.

Diamond-Blackfan anemia syndrome is a ribosomopathy classified among the bone marrow failure syndromes. This disease exhibits significant heterogeneity, with up to 24 genetic variants identified to date. Voit et al.

Right ventricular myocardial work: proof-of-concept for the assessment of pressure-strain loops of patients with pre-capillary pulmonary hypertension.

Background: Right ventricular myocardial work (RVMW) assessed by transthoracic echocardiography allows to study the right ventricular (RV) function using RV pressure-strain loops. The assessment of these novel indexes of RVMW has not yet been exten sively studied, namely in pre-capillary pulmonary hypertension (PH) population.

Objectives: to evaluate the relationship between RVMW and invasive indices of right heart catheterization (RHC) in a cohort of patients with group I and group IV PH and to compare with a control group without PH.

Outcomes of Patients with Heart Failure Hospitalized for COVID-19-A Study in a Tertiary Italian Center.

Coronavirus Disease 2019 (COVID-19), triggered by SARS-CoV-2, has represented a global pandemic associated with an elevated rate of mortality, mainly among older individuals. The extensive pulmonary involvement by the viral infection might have precipitated pre-existing chronic conditions in this vulnerable population, including heart failure (HF). The aim of this retrospective, observational study was to assess the impact of COVID-19 in patients with a prior diagnosis of HF referred to the Emergency Department of the Agostino Gemelli University Hospital between March 2020 and January 2023.

Collagen scaffold-seeded iTenocytes accelerate the healing and functional recovery of Achilles tendon defects in a rat model.

Introduction: Tendon injuries represent an ongoing challenge in clinical practice due to poor regenerative capacity, structure, and biomechanical function recovery of ruptured tendons. This study is focused on the assessment of a novel strategy to repair ruptured Achilles tendons in a Nude rat model using stem cell-seeded biomaterial.

Methods: Specifically, we have used induced pluripotent stem cell (iPSC)-derived mesenchymal stem cells (iMSCs) overexpressing the early tendon marker Scleraxis (SCX, iMSC, iTenocytes) in combination with an elastic collagen scaffold.

Should the Faecal Microbiota Composition Be Determined to Certify a Faecal Donor?

Background/objectives: Faecal microbiota transplantation (FMT) is considered a safe and effective therapy for recurrent infection. It is the only current clinical indication for this technique, although numerous clinical research studies and trials propose its potential usefulness for treating other pathologies. Donor selection is a very rigorous process, based on a personal lifestyle interview and the absence of known pathogens in faeces and serum, leading to only a few volunteers finally achieving the corresponding certification.