Abstract:
:Most lysosomal storage diseases (LSD) exhibit neurological symptoms and there has been limited success in their treatment. Innovative treatments employing novel therapy or gene therapy may offer the prospect of improvement. Recent attempts to treat the neurological forms of LSD include neural stem cell therapy, mesenchymal stem cell therapy, hematopoietic stem cell therapy and gene therapy. Additional approaches have included substrate deprivation/chaperone therapy for the treatment of LSD. This article reviews these new technologies, discusses recent progress, and suggests their possible application.
journal_name
Curr Mol Medjournal_title
Current molecular medicineauthors
Eto Y,Ohashi Tdoi
10.2174/1566524023363112keywords:
subject
Has Abstractpub_date
2002-02-01 00:00:00pages
83-9issue
1eissn
1566-5240issn
1875-5666journal_volume
2pub_type
杂志文章,评审abstract::Visfatin is one of the prominent adipokines secreted by adipose tissue. The level of visfatin increases significantly in persons with obesity owing to increased body mass index (BMI). During obesity, the adipocytes, which populate adipose tissue, undergo hypertrophy and hyperplasia and secrete a number of adipocytokin...
journal_title:Current molecular medicine
pub_type: 杂志文章,评审
doi:10.2174/1566524018666180705114131
更新日期:2018-01-01 00:00:00
abstract::Human induced pluripotent stem cells (hiPSCs) have great potential as a robust source of progenitors for regenerative medicine. The novel technology also enables the derivation of patient-specific cells for applications to personalized medicine, such as for personal drug screening and toxicology. However, the biologic...
journal_title:Current molecular medicine
pub_type: 杂志文章,评审
doi:10.2174/1566524011313050010
更新日期:2013-06-01 00:00:00
abstract::The view that mitochondrial electron transport is the only site of aerobic respiration and the primary bioenergetic pathway in mammalian cells is well established in the literature. Although this paradigm is widely accepted for most tissues, the situation is less clear for proliferating cells. Increasing evidence indi...
journal_title:Current molecular medicine
pub_type: 杂志文章,评审
doi:10.2174/156652406779010777
更新日期:2006-12-01 00:00:00
abstract::Salmonella infections are a serious public health problem in developing countries and represent a constant concern for the food industry. The severity and the outcome of a systemic Salmonella infection depends on the "virulence" of the bacteria, on the infectious dose as well as on the genetic makeup and immunological...
journal_title:Current molecular medicine
pub_type: 杂志文章,评审
doi:10.2174/1566524023362492
更新日期:2002-06-01 00:00:00
abstract:PURPOSE:Protein sumoylation is a well established regulatory mechanism to control many cellular processes such as chromatin structure dynamics, transcriptional regulation of gene expression, cell proliferation and differentiation, cell transformation and carcinogenesis, autophagy and senescence. In the vertebrate visio...
journal_title:Current molecular medicine
pub_type: 杂志文章
doi:10.2174/1566524019666190112143636
更新日期:2018-01-01 00:00:00
abstract::Although GHRH and GHRH-R are recognized as key factors in placental development, little is known about the mechanism(s) of the regulation in trophoblastic cells during placental development. The objective of this study is to determine the potential relationship between the expression levels of GHRH-R and the placental...
journal_title:Current molecular medicine
pub_type: 杂志文章
doi:10.2174/1566524016666160225154040
更新日期:2016-01-01 00:00:00
abstract::Chondroitin sulfate (CS)-glycosaminoglycans (GAGs) are linear, negatively charged polysaccharides attached to CS proteoglycans that make up a major component of biological matrices throughout both central and peripheral tissues. The position of their attached sulfate groups to the CS disaccharide is predicted to influ...
journal_title:Current molecular medicine
pub_type: 杂志文章
doi:10.2174/1566524020666200915110707
更新日期:2020-09-15 00:00:00
abstract::Our current understanding of the mechanisms of information processing and storage in the brain, based on the concept proposed more than fifty years ago by D. Hebb, is that a key role is played by changes in synaptic efficacy induced by coincident pre- and postsynaptic activity. Decades of studies of the properties of ...
journal_title:Current molecular medicine
pub_type: 杂志文章,评审
doi:10.2174/1566524023362041
更新日期:2002-11-01 00:00:00
abstract:BACKGROUND:In this study, the antioxidant property of new synthesized azomethins has been investigated as theoretical and experimental. METHODS AND RESULTS:Density functional theory (DFT) was employed to investigate the Bond Dissociation Enthalpy (BDE), Mulliken Charges, NBO analysis, Ionization Potential (IP), Electr...
journal_title:Current molecular medicine
pub_type: 杂志文章
doi:10.2174/1566524019666190509102620
更新日期:2019-01-01 00:00:00
abstract::The purpose of this review is to provide a concise overview of developments over the last 15 years in the field of laboratory tests in human medicine that are based on the detection of alterations in the glycan part of glycoconjugates. We show how glycosylation-based diagnostic testing is widespread in the current cli...
journal_title:Current molecular medicine
pub_type: 杂志文章,评审
doi:10.2174/156652407780831629
更新日期:2007-06-01 00:00:00
abstract::The generation of functional neural progenitor cells (NPCs) independent of donor brain tissue and embryonic tissues is of great therapeutic interest with regard to regenerative medicine and the possible treatment of neurodegenerative disorders. Traditionally, NPCs are derived through the differentiation of embryonic s...
journal_title:Current molecular medicine
pub_type: 杂志文章
doi:10.2174/156652412798889018
更新日期:2012-02-01 00:00:00
abstract::Many potential HIV vaccine strategies are being explored in both animal model and human settings. The success of any vaccine relies on relevant antigenic determinants being presented to the immune system for the activation of broad and long-lasting immunity. Effective immunity against HIV infection will likely require...
journal_title:Current molecular medicine
pub_type: 杂志文章,评审
doi:10.2174/1566524033479870
更新日期:2003-05-01 00:00:00
abstract::p66Shc is the only known proapoptotic member of the Shc protein family of molecular adaptors. Through its redox activity, p66Shc oxidates cytochrome-c, leading to increased ROS production and, eventually, to apoptosis. p66Shc has been implicated in the control of oxidative stress and life span in mammals. In this revi...
journal_title:Current molecular medicine
pub_type: 杂志文章,评审
doi:10.2174/156652409787847254
更新日期:2009-04-01 00:00:00
abstract::The unexpected encounter between the fields of HIV and chemokines has opened new perspectives for understanding the mechanisms of AIDS pathogenesis, as well as for the development of effective therapies and vaccines. Selected chemokines act as potent natural inhibitors of HIV infection, as they bind and downmodulate c...
journal_title:Current molecular medicine
pub_type: 杂志文章,评审
doi:10.2174/1566524023361862
更新日期:2002-12-01 00:00:00
abstract::Since the discovery almost fifteen years ago that E2F transcription factors are key targets of the retinoblastoma protein (RB), studies of the E2F family have uncovered critical roles in the control of transcription, cell cycle and apoptosis. E2F proteins are encoded by at least eight genes, E2F1 through E2F8. While s...
journal_title:Current molecular medicine
pub_type: 杂志文章,评审
doi:10.2174/1566524010606070739
更新日期:2006-11-01 00:00:00
abstract::Prostate cancer is one of the most difficult cancers to treat especially when it becomes hormone resistant such as castrate resistant prostate cancer (CRPC) and subsequent metastatic CRPC. Apart from the genetic alterations in prostate cancer, epigenetic modifications also play an important role in the development and...
journal_title:Current molecular medicine
pub_type: 杂志文章,评审
doi:10.2174/1566524018666171219101937
更新日期:2017-01-01 00:00:00
abstract::The tumor necrosis factor-related apoptosis-inducing ligand (TRAIL) exerts a cancer cell-specific pro-apoptotic activity. This property made the TRAIL associated pathway one of the most promising strategies aimed at inducing tumor-selective death. In fact, several approaches have been considered to explore this pathwa...
journal_title:Current molecular medicine
pub_type: 杂志文章,评审
doi:10.2174/156652413804810727
更新日期:2013-02-01 00:00:00
abstract::HMGB1/Amphoterin is a ubiquitous, highly conserved DNA-binding protein that can be also released to the extracellular space by various cell types. Extracellular HMGB1 regulates migratory responses of several cell types through binding to RAGE that communicates with the cytoskeleton to regulate cell motility. HMGB1-ind...
journal_title:Current molecular medicine
pub_type: 杂志文章,评审
doi:10.2174/156652407783220750
更新日期:2007-12-01 00:00:00
abstract::Extracellular matrices (ECMs) are dynamic structures that provide cells not only with a structural support but, importantly, exhibit significant functional roles in the control of key cellular events such as adhesion, migration, proliferation, differentiation, and survival. In tumors, matrix effectors such as proteogl...
journal_title:Current molecular medicine
pub_type: 杂志文章,评审
doi:10.2174/156652412802480943
更新日期:2012-09-01 00:00:00
abstract:BACKGROUND:Colorectal cancer (CRC) is the third most common cancer worldwide and the third leading cause of cancer-related death. It is a heterogeneous disease that develops through different genetic and epigenetic mechanisms. To date, no comprehensive systematic review investigating genetic risk factors for familial a...
journal_title:Current molecular medicine
pub_type: 杂志文章
doi:10.2174/1566524019666191014170136
更新日期:2020-01-01 00:00:00
abstract:PURPOSE:To evaluate cytokine expression profile in aqueous humor and sera in patients with HLAB27 associated acute anterior uveitis (AAU) and idiopathic AAU. METHODS:Twenty patients with AAU and 17 controls were recruited from August 2012 to March 2013. Study subjects with uveitis were divided into two groups: 9 patie...
journal_title:Current molecular medicine
pub_type: 杂志文章
doi:10.2174/1566524015666150731100012
更新日期:2015-01-01 00:00:00
abstract::Non-alcoholic fatty liver disease (NAFLD) is a clinico-pathologic spectrum of conditions ranging from simple steatosis to nonalcoholic steatohepatitis (NASH). Although simple or bland steatosis follows a relatively benign clinical course, NASH can potentially progress to cirrhosis (approximately 10 to 15 percent) and ...
journal_title:Current molecular medicine
pub_type: 杂志文章,评审
doi:10.2174/156652410790963321
更新日期:2010-03-01 00:00:00
abstract:PURPOSE:Ubiquitin is involved in cell proliferation and differentiation, and the objective of this study is to investigate the potential of dominant negative Ubiquitin (Ub) with a lysine to tryptophan mutation at the 6 position (K6W) through an adenoviral expression vector in the prevention of posterior capsule opacifi...
journal_title:Current molecular medicine
pub_type: 杂志文章
doi:10.2174/1566524017666170331163751
更新日期:2017-01-01 00:00:00
abstract::Despite advances in biomedical sciences, the prognosis of patients with brain tumors remains poor. Effective treatment is lacking for these central nervous system (CNS) cancers. Targeted immunotoxins are a new class of therapeutic approaches that have emerged for the treatment of human cancers. In this approach, tumor...
journal_title:Current molecular medicine
pub_type: 杂志文章,评审
doi:10.2174/156652409788970661
更新日期:2009-08-01 00:00:00
abstract::Skin is a complex tissue composed of two very different compartments -- the continuously renewing epidermis made up mostly by keratinocytes and the underlying matrix-rich dermis with the resting fibroblasts as its major cellular components. Both compartments are tightly interconnected and a paracrine mutual interactio...
journal_title:Current molecular medicine
pub_type: 杂志文章,评审
doi:10.2174/1566524053586644
更新日期:2005-03-01 00:00:00
abstract::As tumors progress to increased malignancy, cells within them develop the ability to invade into surrounding normal tissues and through tissue boundaries to form new growths (metastases) at sites distinct from the primary tumor. The molecular mechanisms involved in this process are incompletely understood but those as...
journal_title:Current molecular medicine
pub_type: 杂志文章,评审
doi:10.2174/1566524033479447
更新日期:2003-11-01 00:00:00
abstract:BACKGROUND & OBJECTIVE:Interleukin 37 (IL-37) is an important regulator of the anti-inflammatory T-cell response. In this study, we investigated its expression and function in peripheral blood mononuclear cells (PBMCs) of patients with HLA-B27 associated acute anterior uveitis (AAU) and idiopathic AAU. METHODS:15 pati...
journal_title:Current molecular medicine
pub_type: 杂志文章
doi:10.2174/1566524018666180207152941
更新日期:2018-03-09 00:00:00
abstract::Similar to the human immunodeficiency virus (HIV), the hepatitis B virus (HBV) replicates via reverse transcription, in this case, within infected hepatocytes. Substantial advances have been achieved in the past ten years in developing and utilizing nucleoside/nucleotide analog drugs to inhibit HBV replication. Most a...
journal_title:Current molecular medicine
pub_type: 杂志文章,评审
doi:10.2174/156652407780059159
更新日期:2007-03-01 00:00:00
abstract::Abstract: The study of genetic alterations in tumors and their precursor lesions is often hampered by the presence of a heterogeneous background of non-neoplastic elements such as stromal cells, inflammatory cells, and angiogenic elements. Microdissection involves the extraction of specific populations of cells under ...
journal_title:Current molecular medicine
pub_type: 杂志文章,评审
doi:10.2174/1566524013364121
更新日期:2001-03-01 00:00:00
abstract::Classical Hodgkin lymphoma (cHL) is now recognized as a B-cell-derived lymphoma which is characterized by only about 1% malignant pathognomonic Hodgkin and Reed-Sternberg (HRS) cells and an abundant infiltrate of reactive bystander cells. HRS cells are unique with respect to their lost B-cell-specific gene expression ...
journal_title:Current molecular medicine
pub_type: 杂志文章,评审
doi:10.2174/156652411795243423
更新日期:2011-04-01 00:00:00