Stem Cell Research Tools in Human Metabolic Disorders: An Overview

Abstract

Metabolic disorders are very common in the population worldwide and are among the diseases with the highest health utilization and costs per person. Despite the ongoing efforts to develop new treatments, currently, for many of these disorders, there are no approved therapies, resulting in a huge economic hit and tension for society. In this review, we recapitulate the recent advancements in stem cell (gene) therapy as potential tools for the long-term treatment of both inherited (lysosomal storage diseases) and acquired (diabetes mellitus, obesity) metabolic disorders, focusing on the main promising results observed in human patients and discussing the critical hurdles preventing the definitive jump of this approach from the bench to the clinic.

Cellular microenvironment: a key for tuning mesenchymal stem cell senescence

“…Inborn metabolic disorders encompass various types with complex metabolic defects, and a perfect classification method has yet to be established. Previous research has primarily focused on experimental studies of stem cell therapy for inborn metabolic disorders related to the connective tissue, musculoskeletal, neurological, and hematopoietic systems ( Ricci and Cacialli, 2021 ; Specchio et al, 2021 ). For instance, MSCs and their EVs have shown potential in treating corneal diseases caused by Mucopolysaccharidoses by delivering the enzyme N-acetylgalactosamine-6-sulfate sulfatase (GALNS) to defective cells ( Flanagan et al, 2021 ).…”

Hematopoietik Kök Hücre Nakil Süreci

“…Nakil tekniklerindeki ve destekleyici bakım stratejilerindeki ilerlemeler, hayatta kalma oranlarında önemli gelişmelere olanak sağlamıştır 26 . Kanserler (örneğin lösemi ve lenfoma), kalp yetmezliği, sinir bozuklukları, otoimmün hastalıklar, metabolizma hastalıkları (örneğin diabetes mellitus, obezite ve osteoporoz) veya genetik bozukluklar (örneğin lizozomal depo hastalığı, peroksizomal hastalıklar ve mitokondriyal hastalıklar) gibi çeşitli hastalıkları tedavi etmek için kök hücre nakli gerçekleştirilmektedir 27,28 . Kök hücre nakil çeşitleri; kök hücre kaynağına göre (kemik iliği kök hücre nakli, periferik kan kök hücre nakli ve kordon kanı kök hücre nakli) veya donör tipine göre (otolog kök hücre nakli ve allojenik kök hücre nakli) veya hazırlama rejimine göre (miyeloablatif kök hücre nakli ve miyeloablatif olmayan kök hücre nakli) sınıflandırılabilir 5,29 .…”

Transcription Pattern of Neurotrophic Factors and Their Receptors in Adult Zebrafish Spinal Cord

“…In the spinal cord, neurotrophins and receptors can modulate repair after a traumatic event [ 19 , 20 ]. Indeed, recent functional in vivo studies in mouse and rat models showed that cell transplants [ 21 ], including engineered stem cells (that overexpress neurotrophins and/or receptors), can affect the function of spinal neurons and circuits [ 22 ]. However, further studies using emerging animal models, such as the zebrafish ( Danio rerio ), could be useful to elucidate the specific mechanisms of this damage.…”