Regulatory systems and requirements for clinical trials of AAV-based gene therapies - Perspectives from six Asian countries or regions: Report from the 6th Asia Partnership Conference of Regenerative Medicine - April 20, 2023.

Gene therapies, which include viral-vector gene delivery, genome editing, and genetically modified cell therapy, are innovative treatments with the potential to address the underlying genetic causes of disorders and to provide life-changing value in terms of curing disease. Although adeno-associated virus (AAV)-based gene therapy is one of the most advanced types of gene therapy, far fewer AAV-based gene therapy studies have been conducted in Asia than in North America and Europe. The 6th Asia Partnership Conference of Regenerative Medicine (APACRM) was held on April 20, 2023 in Tokyo, Japan.

Advantages of pooling of human bone marrow-derived mesenchymal stromal cells from different donors versus single-donor MSCs.

Mesenchymal stromal cells (MSC) from adult bone marrow are the most commonly used cells in clinical trials. MSCs from single donors are the preferred starting material but suffer from a major setback of being heterogeneous that results in unpredictable and inconsistent clinical outcomes. To overcome this, we developed a method of pooling MSCs from different donors and created cell banks to cater clinical needs.

Label extension, single-arm, phase III study shows efficacy and safety of stempeucel® in patients with critical limb ischemia due to atherosclerotic peripheral arterial disease.

Background: Peripheral arterial disease (PAD) of lower extremities comprises a clinical spectrum that extends from asymptomatic patients to critical limb ischemia (CLI) patients. 10% to 40% of the patients are at the risk of primary amputation. This study was planned in "no-option" patients of CLI due to atherosclerotic PAD to assess the efficacy and safety of pooled, allogeneic, adult human bone marrow-derived mesenchymal stromal cells which is already approved for marketing in India for CLI due to Buerger's disease.

Effect of Enteral Long-Chain Polyunsaturated Fatty Acids on Retinopathy of Prematurity: A Systematic Review and Meta-Analysis.

Background: Long-chain polyunsaturated fatty acids (LCPUFA) are critical for the maturation of the brain and retina. Retinopathy of prematurity (ROP) is a preventable cause of blindness in preterm infants. LCPUFA have anti-inflammatory, antioxidant, and antiangiogenesis effects.

Pituitary Tuberculoma Masquerading as a Pituitary Adenoma: Interesting Case Report and Review of Literature.

We present a rare case of a pituitary tuberculoma masquerading as pituitary adenoma with pituitary apoplexy-like presentation in a 31-year-old female, who had symptoms suggestive of acute secondary adrenal insufficiency with secondary amenorrhea. After initial evaluation which was suggestive of pituitary adenoma, she underwent endoscopic transnasal pituitary tumor excision. Histopathology revealed features of pituitary tuberculoma.

Association Between Non-Cystic Fibrosis Bronchiectasis and Quality of Life: A Single-Center Cross-Sectional Study.

Introduction Bronchiectasis is a chronic respiratory disease that can affect patients of all ages and significantly impact the quality of life (QOL) in patients who suffer from it. In spite of its widespread prevalence, and the significant impact on QOL, data on the quantitative impact of bronchiectasis on QOL is lacking. The Quality of Life-Bronchiectasis (QOL-B) is a self-administered patient-reported outcome measure, that was recently developed as a response to the emergent need for such measurement tools to study the impact of bronchiectasis on QOL.