scholarly journals Stem Cell Therapy for the Central Nervous System in Lysosomal Storage Diseases

2016 ◽  
Vol 27 (10) ◽  
pp. 749-757 ◽  
Author(s):  
Faez Siddiqi ◽  
John H. Wolfe
Keyword(s):  
Central Nervous System ◽  
Nervous System ◽  
Stem Cell ◽  
Cell Therapy ◽  
Stem Cell Therapy ◽  
Lysosomal Storage Diseases ◽  
Lysosomal Storage ◽  
Storage Diseases ◽  
The Central Nervous System

scholarly journals Immunological Barriers to Stem Cell Therapy in the Central Nervous System

2014 ◽  
Vol 2014 ◽  
pp. 1-12 ◽  
Author(s):  
Gregory E. Tullis ◽  
Kathleen Spears ◽  
Mark D. Kirk
Keyword(s):  
Central Nervous System ◽  
Nervous System ◽  
Stem Cell ◽  
Cell Therapy ◽  
Stem Cell Therapy ◽  
Brain Disorders ◽  
Stem Cell Therapies ◽  
Cell Therapies ◽  
Cell Transplants ◽  
The Central Nervous System

The central nervous system is vulnerable to many neurodegenerative disorders such as Alzheimer’s disease that result in the extensive loss of neuronal cells. Stem cells have the ability to differentiate into many types of cells, which make them ideal for treating such disorders. Although stem cell therapy has shown some promising results in animal models for many brain disorders it has yet to translate into the clinic. A major hurdle to the translation of stem cell therapy into the clinic is the immune response faced by stem cell transplants. Here, we focus on immunological and related hurdles to stem cell therapies for central nervous system disorders.

scholarly journals New paradigms for the treatment of lysosomal storage diseases: targeting the endocannabinoid system as a therapeutic strategy

2021 ◽  
Vol 16 (1) ◽  
Author(s):  
Edward H. Schuchman ◽  
Maria D. Ledesma ◽  
Calogera M. Simonaro
Keyword(s):  
Central Nervous System ◽  
Nervous System ◽  
Endocannabinoid System ◽  
Lysosomal Storage Diseases ◽  
Common Disease ◽  
Lysosomal Storage ◽  
Treatment Development ◽  
Storage Diseases ◽  
Lysosomal Diseases ◽  
The Central Nervous System

AbstractOver the past three decades the lysosomal storage diseases have served as model for rare disease treatment development. While these efforts have led to considerable success, important challenges remain. For example, no treatments are currently approved for nearly two thirds of all lysosomal diseases, and there is limited impact of the existing drugs on the central nervous system. In addition, the costs of these therapies are extremely high, in part due to the fact that drug development has focused on a “single hit” approach – i.e., one drug for one disease. To overcome these obstacles researchers have begun to focus on defining common disease mechanisms in the lysosomal diseases, particularly in the central nervous system, with the hope of identifying drugs that might be used in several lysosomal diseases rather than an individual disease. With this concept in mind, herein we review a new potential treatment approach for the lysosomal storage diseases that focuses on modulation of the endocannabinoid system. We provide a short introduction to lysosomal storage diseases and the endocannabinoid system, followed by a brief review of data supporting this concept.

scholarly journals Lysosomal Storage Diseases-Regulating Neurodegeneration

2015 ◽  
Vol 9s2 ◽  
pp. JEN.S25475 ◽  
Author(s):  
Rob U. Onyenwoke ◽  
Jay E. Brenman
Keyword(s):  
Central Nervous System ◽  
Nervous System ◽  
Peripheral Nervous System ◽  
Pompe Disease ◽  
Lysosomal Storage Diseases ◽  
Cell Types ◽  
Lysosomal Storage ◽  
Storage Diseases ◽  
The Central Nervous System ◽  
Made In

Autophagy is a complex pathway regulated by numerous signaling events that recycles macromolecules and can be perturbed in lysosomal storage diseases (LSDs). The concept of LSDs, which are characterized by aberrant, excessive storage of cellular material in lysosomes, developed following the discovery of an enzyme deficiency as the cause of Pompe disease in 1963. Great strides have since been made in better understanding the biology of LSDs. Defective lysosomal storage typically occurs in many cell types, but the nervous system, including the central nervous system and peripheral nervous system, is particularly vulnerable to LSDs, being affected in two-thirds of LSDs. This review provides a summary of some of the better characterized LSDs and the pathways affected in these disorders.

Regenerative medicine for central nervous system disorders: Role of therapeutic molecules in stem cell therapy

2015 ◽  
Vol 1 (2) ◽  
pp. 125 ◽  
Author(s):  
CesarioV Borlongan ◽  
Paola Suárez-Meade ◽  
HoracioG Carvajal ◽  
Takao Yasuhara ◽  
Naoki Tajiri ◽  
...  
Keyword(s):  
Central Nervous System ◽  
Nervous System ◽  
Stem Cell ◽  
Regenerative Medicine ◽  
Cell Therapy ◽  
Stem Cell Therapy ◽  
Central Nervous System Disorders ◽  
Therapeutic Molecules
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