Regulated hAAT Expression from a Novel rAAV Vector and Its Application in the Prevention of Type 1 Diabetes

scientific article published on 28 August 2019

Regulated hAAT Expression from a Novel rAAV Vector and Its Application in the Prevention of Type 1 Diabetes is …
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scholarly articleQ13442814

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P356DOI10.3390/JCM8091321
P932PMC publication ID6780368
P698PubMed publication ID31466263

P50authorHong-Xia MaQ87735703
P2093author name stringClive Wasserfall
Sihong Song
Mark A Atkinson
Yuanqing Lu
Keith Lowe
Lonneke van der Meijden-Erkelens
P2860cites workAdeno-associated virus vector as a platform for gene therapy deliveryQ64040883
Both the Lyt-2+ and L3T4+ T cell subsets are required for the transfer of diabetes in nonobese diabetic miceQ68123525
Autoimmune diabetes in NOD mouse is L3T4 T-lymphocyte dependentQ70327797
Recombinant adeno-associated viral vectors mediate long-term transgene expression in muscleQ73254110
Comment on: Brugman S et al. (2006) Antibiotic treatment partially protects against type 1 diabetes in the Bio-Breeding diabetes-prone rat. Is the gut flora involved in the development of type 1 diabetes? Diabetologia 49:2105-2108Q79375363
Antibiotic treatment partially protects against type 1 diabetes in the Bio-Breeding diabetes-prone rat. Is the gut flora involved in the development of type 1 diabetes?Q79830050
Type 1 diabetes: focus on preventionQ80402893
Alpha 1 Antitrypsin Gene Therapy Extends the Lifespan of Lupus-Prone MiceQ90468943
Alpha-1 Antitrypsin Therapy for Autoimmune DisordersQ91363611
Alpha1-antitrypsin protects beta-cells from apoptosisQ40158851
Alpha-1 Antitrypsin Gene Therapy Ameliorates Bone Loss in Ovariectomy-Induced Osteoporosis Mouse ModelQ40763831
The circulating proteinase inhibitor α-1 antitrypsin regulates neutrophil degranulation and autoimmunity.Q42234561
Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brainQ42280576
Anti-inflammaging effects of human alpha-1 antitrypsinQ42778200
Combination of alpha-1 antitrypsin and doxycycline suppresses collagen-induced arthritisQ42832630
Intradermal alpha1-antitrypsin therapy avoids fatal anaphylaxis, prevents type 1 diabetes and reverses hyperglycaemia in the NOD mouse model of the diseaseQ42996820
Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectorsQ43716886
Efficient and stable adeno-associated virus-mediated transduction in the skeletal muscle of adult immunocompetent miceQ44678756
Recombinant adeno-associated virus-mediated alpha-1 antitrypsin gene therapy prevents type I diabetes in NOD miceQ44719693
Autoimmune diabetes and the circle of toleranceQ44745115
Alpha-1 antitrypsin inhibits caspase-1 and protects from acute myocardial ischemia-reperfusion injuryQ44966048
Therapeutic level of functional human alpha 1 antitrypsin (hAAT) secreted from murine muscle transduced by adeno-associated virus (rAAV1) vectorQ45419136
Stable therapeutic serum levels of human alpha-1 antitrypsin (AAT) after portal vein injection of recombinant adeno-associated virus (rAAV) vectorsQ45735547
Alpha1-antitrypsin gene therapy modulates cellular immunity and efficiently prevents type 1 diabetes in nonobese diabetic miceQ45859046
CMV-beta-actin promoter directs higher expression from an adeno-associated viral vector in the liver than the cytomegalovirus or elongation factor 1 alpha promoter and results in therapeutic levels of human factor X in miceQ45875988
NKG2D blockade prevents autoimmune diabetes in NOD miceQ46294168
T-lymphocyte clone specific for pancreatic islet antigenQ46335815
Human alpha 1-antitrypsin therapy induces fatal anaphylaxis in non-obese diabetic mice.Q46401098
Gene regulation of the serine proteinase inhibitors alpha1-antitrypsin and alpha1-antichymotrypsinQ28218698
Alpha 1-antitrypsin therapy mitigated ischemic stroke damage in ratsQ33671373
Alpha1-antitrypsin monotherapy prolongs islet allograft survival in miceQ33922982
Adeno-associated virus vector-mediated IL-10 gene delivery prevents type 1 diabetes in NOD miceQ33950195
The discovery of α1-antitrypsin and its role in health and diseaseQ34025923
Gene- and cell-based therapeutics for type I diabetes mellitusQ35122170
alpha-1 antitrypsin inhibits caspase-3 activity, preventing lung endothelial cell apoptosisQ35607245
Alpha-1-antitrypsin monotherapy reduces graft-versus-host disease after experimental allogeneic bone marrow transplantationQ35673668
Role of caspases in the regulation of apoptotic pancreatic islet beta-cells death.Q35790490
Pancreas transplantation. An immunohistologic and histopathologic examination of 100 grafts.Q35856482
Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vectorQ35872651
Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic proteinQ35943644
Alpha 1 Antitrypsin Inhibits Dendritic Cell Activation and Attenuates Nephritis in a Mouse Model of LupusQ36032325
Efficient photoreceptor-targeted gene expression in vivo by recombinant adeno-associated virusQ36242842
Prevention of diabetes in BioBreeding/Worcester rats with monoclonal antibodies that recognize T lymphocytes or natural killer cellsQ36352899
Syngeneic transfer of autoimmune diabetes from diabetic NOD mice to healthy neonates. Requirement for both L3T4+ and Lyt-2+ T cells.Q36353954
In vitro-expanded antigen-specific regulatory T cells suppress autoimmune diabetesQ36399048
CD25+ CD4+ T cells, expanded with dendritic cells presenting a single autoantigenic peptide, suppress autoimmune diabetes.Q36399104
Stable in vivo expression of the cystic fibrosis transmembrane conductance regulator with an adeno-associated virus vectorQ36663364
Sustained secretion of human alpha-1-antitrypsin from murine muscle transduced with adeno-associated virus vectorsQ36734619
alpha1-Antitrypsin monotherapy induces immune tolerance during islet allograft transplantation in miceQ36935759
Safety and efficacy of alpha-1-antitrypsin augmentation therapy in the treatment of patients with alpha-1-antitrypsin deficiency.Q37303247
Distinct immune responses to transgene products from rAAV1 and rAAV8 vectorsQ37385999
Tight Long-term dynamic doxycycline responsive nigrostriatal GDNF using a single rAAV vector.Q39502530
Adeno-associated virus as a vector for liver-directed gene therapy.Q39583470
Alpha-1 antitrypsin protein and gene therapies decrease autoimmunity and delay arthritis development in mouse modelQ39587262
P275copyright licenseCreative Commons Attribution 4.0 InternationalQ20007257
P6216copyright statuscopyrightedQ50423863
P433issue9
P921main subjecttype-1 diabetesQ124407
P577publication date2019-08-28
P1433published inJournal of Clinical MedicineQ27724774
P1476titleRegulated hAAT Expression from a Novel rAAV Vector and Its Application in the Prevention of Type 1 Diabetes
P478volume8