Abstract:
:Spinocerebellar ataxia type 1 (SCA1) is a dominant inherited disease caused by expanded trinucleotide repeats resulting in an increased polyglutamine tract in the gene product. As a potential therapeutic approach for SCA1, we tested antisense RNAs targeting two regions of the ataxin-1 message. Single-stranded regions around the translational initiation site and the intron 8 splice donor site of the ataxin-1 message were identified by computer-assisted RNA secondary structure prediction. Plasmids were generated to contain a 254-bp antisense sequence spanning the translation initiation site (pLasBDini) or a 317-bp sequence spanning the intron 8 splice donor site (pLasBDei) of the ataxin-1 message. These plasmids were transfected into Chinese hamster ovary cells engineered to express either expanded or unexpanded ataxin-1 message and protein. Reduced levels of mutant ataxin-1 message (82 CAG repeats), wild-type ataxin-1 message (30 CAG repeats), and ataxin-1 protein were observed by Northern and Western blot analyses in pLasBDini-transfected clones. pLasBDei-transfected 293 cells exhibited a shift in ataxin-1 message to a size several kilobases longer than that of the natural message. Reverse transcriptase/polymerase chain reaction assays demonstrated the retention of message spanning the intron 8 splice acceptor and the inability to amplify sequences between exons 8 and 9, implying that normal splicing of intron 8 had been interrupted. We conclude that antisense RNAs were effective in reducing or modifying ataxin-1 messages in transfected cells, and may be an effective genetic strategy for therapy of SCA1 and similar dominant-acting neurological disorders.
journal_name
Cell Transplantjournal_title
Cell transplantationauthors
Gao Y,Zu T,Low WC,Orr HT,McIvor RSdoi
10.3727/096368908786516729subject
Has Abstractpub_date
2008-01-01 00:00:00pages
723-34issue
7eissn
0963-6897issn
1555-3892journal_volume
17pub_type
杂志文章abstract::The mechanisms of donor hepatocyte integration into recipient liver are not fully understood. We investigated mechanisms of both the integration and interaction of transplanted hepatocytes with host liver cells as well as the repopulation of the host organ following intraportal transplantation. Mature hepatocytes were...
journal_title:Cell transplantation
pub_type: 杂志文章
doi:10.3727/000000005783983322
更新日期:2005-01-01 00:00:00
abstract::For clinical islet transplantation, isolated islets deteriorate rapidly in culture, although culturing islets prior to transplantation provides flexibility for evaluation of isolated islets and pretreatment of patients. In the present study, we compared human fresh islets to cultured islets with in vitro and in vivo ...
journal_title:Cell transplantation
pub_type: 杂志文章
doi:10.3727/096368911X605439
更新日期:2012-01-01 00:00:00
abstract::Stem cell transplantation represents a promising therapeutic approach for myocardial ischemia/reperfusion (I/R) injury, where cortical bone-derived stem cells (CBSCs) stand out and hold superior cardioprotective effects on myocardial infarction than other types of stem cells. However, the molecular mechanism underlyin...
journal_title:Cell transplantation
pub_type: 杂志文章
doi:10.1177/0963689718786882
更新日期:2018-08-01 00:00:00
abstract::Successful repair of damage in cerebral palsy (CP) needs effective clinical interventions other than simply symptomatic treatments. To elucidate the feasibility of using olfactory ensheathing cells (OECs) to treat CP in children and adolescents, we conducted a randomized controlled clinical trial (RCT) on 33 patients....
journal_title:Cell transplantation
pub_type: 杂志文章,随机对照试验
doi:10.3727/096368910X492652
更新日期:2010-01-01 00:00:00
abstract::Embedded-culture of pancreatic beta-cells derived from a transgenic mouse insulinoma (MIN6 cells) was studied in vitro and in vivo. The MIN6 cells were enmeshed in an agarose-PVMA-collagen matrix for long-term maintenance. The cells formed islet-like cell clusters (ICCs) in the mixed matrix. When 10 mmol/L nicotinamid...
journal_title:Cell transplantation
pub_type: 杂志文章
doi:10.1016/0963-6897(95)00001-e
更新日期:1995-05-01 00:00:00
abstract::Traumatic brain injury (TBI), a major contributor to deaths and permanent disability worldwide, has been recently described as a progressive cell death process rather than an acute event. TBI pathophysiology is complicated and can be distinguished by the initial primary injury and the subsequent secondary injury that ...
journal_title:Cell transplantation
pub_type: 杂志文章,评审
doi:10.3727/096368915X686913
更新日期:2015-01-01 00:00:00
abstract::Regenerative medicine is a new field primarily based on the concept of transplanting exogenous or stimulating endogenous stem cells to generate biological substitutes and improve tissue functions. Recently, amnion-derived cells have been reported to have multipotent differentiation ability, and these cells have attrac...
journal_title:Cell transplantation
pub_type: 杂志文章,评审
doi:10.3727/096368909788341243
更新日期:2009-01-01 00:00:00
abstract::"Proof-of-principle" that cell replacement therapy works for neurodegeneration has been reported, but only using donor cells collected from fetal brain tissue obtained from surgical terminations of pregnancy. Surgical terminations of pregnancy represent an increasingly limited supply of donor cells due to the tendenc...
journal_title:Cell transplantation
pub_type: 杂志文章
doi:10.3727/096368910X546580
更新日期:2011-01-01 00:00:00
abstract::We have recently reported that small-sized bone marrow cells (BMCs) isolated by counterflow centrifugal elutriation and depleted of lineage markers (Fr25lin(-)) have the capacity to differentiate and contribute to regeneration of injured islets. In this study, we assess some of the characteristics of these cells compa...
journal_title:Cell transplantation
pub_type: 杂志文章
doi:10.3727/096368913X676899
更新日期:2015-01-01 00:00:00
abstract::To engineer liver tissues with a clinically significant size, in vivo evaluation of performance using large-scale animal studies are necessary before proceeding to human clinical trials. As pigs are the most suitable candidates, the development of culture conditions suitable for porcine hepatocyte progenitors is very ...
journal_title:Cell transplantation
pub_type: 杂志文章
doi:10.3727/000000006783981486
更新日期:2006-01-01 00:00:00
abstract::The anatomical specificity of axon growth from fetal pig septal xenografts was studied by transplanting septal cells from E30-35 pig fetuses into cholinergic deafferented (192-IgG-saporin-infused) rats or into aged rats (> 18 months). Cell suspensions (100,000 cells/microl) were injected bilaterally into the dorsal an...
journal_title:Cell transplantation
pub_type: 杂志文章
doi:10.1177/096368979900800104
更新日期:1999-01-01 00:00:00
abstract::Mononuclear stem cells have been studied for their potential in myocardial ischemia. In our previous published article, ReACT(®) phase I/II clinical trial, our results suggest that a certain cell population, promonocytes, directly correlated with the perceived angiogenesis in refractory angina patients. This study is ...
journal_title:Cell transplantation
pub_type: 杂志文章
doi:10.3727/096368914X681595
更新日期:2015-01-01 00:00:00
abstract::Multilineage-differentiating stress-enduring (Muse) cells are endogenous pluripotent stem cells that can be isolated based on stage-specific embryonic antigen-3 (SSEA-3), a pluripotent stem cell-surface marker. However, their capacities for survival, neurotrophic factor secretion, and neuronal and glial differentiatio...
journal_title:Cell transplantation
pub_type: 杂志文章
doi:10.1177/0963689719863809
更新日期:2019-09-01 00:00:00
abstract::Testis-derived Sertoli cells have been used to create an immune "privileged" site outside of the testis to facilitate cell transplantation protocols for diabetes and neurodegenerative diseases. In addition to secreting immunoprotective factors, Sertoli cells also secrete growth and trophic factors that appear to enhan...
journal_title:Cell transplantation
pub_type: 杂志文章
doi:10.1016/s0963-6897(97)00022-5
更新日期:1997-03-01 00:00:00
abstract::Cotransplantation of neural progenitors (NPs) with Schwann cells (SCs) might be a way to overcome low rate of neuronal differentiation of NPs following transplantation in spinal cord injury (SCI) and the improvement of locomotor recovery. In this study, we initially generated NPs from human embryonic stem cells (hESC...
journal_title:Cell transplantation
pub_type: 杂志文章
doi:10.3727/096368911X593163
更新日期:2012-01-01 00:00:00
abstract::Although cells isolated from fetal liver are one of the major sources for liver tissue engineering, it is still very difficult to induce them to fully differentiate in vitro into mature hepatocytes. We therefore investigated the effects of nicotinamide (NA), dimethyl sulfoxide (DMSO), and oncostatin M (OSM) on differe...
journal_title:Cell transplantation
pub_type: 杂志文章
doi:
更新日期:2002-01-01 00:00:00
abstract::Alzheimer disease (AD) is a progressive neurodegenerative disease, which is characterized by loss of memory and cognitive function. In AD patients dysfunction of the cholinergic system is the main cause of cognitive disorders, and decreased activity of choline acetyltransferase (ChAT), an enzyme responsible for acety...
journal_title:Cell transplantation
pub_type: 杂志文章
doi:10.3727/096368911X586765
更新日期:2012-01-01 00:00:00
abstract::Persistent corneal wounds result from numerous eye disorders, and to date, available treatments often fail to accelerate reepithelialization, the key initial step in wound healing. To speed reepithelialization, we explored a cell-transfer transplant method utilizing polydimethylsiloxane (PDMS) contact lenses to delive...
journal_title:Cell transplantation
pub_type: 杂志文章
doi:10.3727/000000005783982783
更新日期:2005-01-01 00:00:00
abstract::The major limitation of nonhuman primate (NHP) embryonic stem (ES) cell research is inefficient genetic modification and limited knowledge of differentiation mechanisms. A genetically modified NHP-ES cell with biomarkers, such as green fluorescent protein (GFP), that allow noninvasive monitoring of transgenic cells, i...
journal_title:Cell transplantation
pub_type: 杂志文章
doi:10.3727/000000002783985350
更新日期:2002-01-01 00:00:00
abstract::Hepatocyte transplantation is dependent on the availability of good quality human hepatocytes isolated from donor liver tissue. Hepatocytes obtained from livers rejected for transplantation on the grounds of steatosis are often of low viability and not suitable for clinical use. The aim of this study was to evaluate t...
journal_title:Cell transplantation
pub_type: 杂志文章
doi:10.3727/096368910X514620
更新日期:2010-01-01 00:00:00
abstract::Ten mongrel dogs were used in this study. Diabetes was chemically induced in 7 dogs, and 3 dogs served as normal controls. For each diabetic dog, 5 million human bone marrow-derived mesenchymal stem cells/kg were differentiated to form insulin-producing cells using a trichostatin-based protocol. Cells were then loaded...
journal_title:Cell transplantation
pub_type: 杂志文章
doi:10.1177/0963689718759913
更新日期:2018-06-01 00:00:00
abstract::Prior studies have suggested the possibility of immune-mediated death of xenogeneic hepatocytes in a bioartificial liver (BAL) during hemoperfusion. This study was designed to elucidate how immunity may cause death of xenogeneic hepatocytes in the BAL. Healthy dogs were treated with a BAL containing hollow fiber membr...
journal_title:Cell transplantation
pub_type: 杂志文章
doi:10.3727/000000004783983378
更新日期:2004-01-01 00:00:00
abstract::The incidence of cell transplant-transmitted infection is unknown and can only be inferred from prospective studies--that have not yet been performed and reported. The possibility of donor-to-recipient disease transmission through cell transplant therapy can be considered by reviewing the risk associated with other tr...
journal_title:Cell transplantation
pub_type: 杂志文章,评审
doi:10.1016/0963-6897(95)00035-v
更新日期:1995-09-01 00:00:00
abstract::Acute myocardial infarction (AMI) results in ischemic damage and death of cardiomyocytes and loss of vasculature. Stem cell therapy has emerged as a potentially promising strategy for maximizing cardiac function following ischemic injury. Issues of cell source, delivery, and quantification of response have challenged...
journal_title:Cell transplantation
pub_type: 杂志文章
doi:10.3727/096368911X603657
更新日期:2012-01-01 00:00:00
abstract::The transplantation of porcine islets of Langerhans to treat type 1 diabetes may provide a solution to the demand for insulin-producing cells. Porcine islets encapsulated in agarose-agarose macrobeads have been shown to function in nonimmunosuppressed xenogeneic models of both streptozotocin-induced and autoimmune typ...
journal_title:Cell transplantation
pub_type: 杂志文章
doi:10.3727/096368913X667033
更新日期:2014-01-01 00:00:00
abstract::In multiple sclerosis (MS), a chronic inflammatory relapsing demyelinating disease, failure to control or repair damage leads to progressive neurological dysfunction and neurodegeneration. Implantation of neural stem cells (NSCs) has been shown to promote repair and functional recovery in the acute experimental autoi...
journal_title:Cell transplantation
pub_type: 杂志文章
doi:10.3727/096368911X637443
更新日期:2012-01-01 00:00:00
abstract::Cell transplantation is now being explored as a new therapeutic strategy to restore function in the diseased human central nervous system. Neural grafts show long-term survival and function in patients with Parkinson's disease but the symptomatic relief needs to be increased. Cell transplantation seems justified in pa...
journal_title:Cell transplantation
pub_type: 杂志文章,评审
doi:10.1016/0963-6897(95)00022-p
更新日期:1995-07-01 00:00:00
abstract::The transplantation of encapsulated dopamine-secreting cells into the striatum represents one potential means of treating Parkinson's disease. The present study investigated the ability of encapsulated PC12 cells, which are derived from rat pheochromocytoma, to supply L-dopa and dopamine into the primate brain in the ...
journal_title:Cell transplantation
pub_type: 杂志文章
doi:10.1177/096368970000900519
更新日期:2000-09-01 00:00:00
abstract::The effect of tensile stress on the orientation and phenotype of arterial smooth muscle cells (SMCs) cultured in three-dimensional (3D) type I collagen gels was morphologically investigated. Ring-shaped hybrid tissues were prepared by thermal gelation of a cold mixed solution of type I collagen and SMCs derived from b...
journal_title:Cell transplantation
pub_type: 杂志文章
doi:10.1177/096368979400300605
更新日期:1994-11-01 00:00:00
abstract::Heart failure continues to be a major health care concern with relatively few options for severely advanced heart failure patients. The hallmark of heart failure is the progressive dilatation of the left ventricle, thinning of the left ventricular wall leading to increased wall stress and increased myocardial oxygen c...
journal_title:Cell transplantation
pub_type: 临床试验,杂志文章
doi:10.3727/096368911X637461
更新日期:2013-01-01 00:00:00