Oral gene therapy for hypoparathyroidism: a rat model.

abstract：:Oncolytic viruses (OV) are novel cancer gene therapies that are moving toward the forefront of modern medicines. However, their full therapeutic potential is hindered by the lack of convenient and reliable strategies to visualize and quantify OV growth kinetics and therapeutic efficacy in live cells. Here, we present ...

journal_title：Human gene therapy

authors： Quillien L,Top S,Kappler S,Redouté A,Dusetti N,Quentin-Froignant C,Lulka H,Camus C,Buscail L,Gallardo F,Bertagnoli S,Cordelier P

更新日期：2021-01-27 00:00:00

abstract：:Diabetes mellitus derives from either insulin deficiency (type I) or resistance (type II). Homozygous mutations in the insulin receptor (IR) gene cause the rare leprechaunism and Rabson-Mendenhall syndromes, severe forms of hyperinsulinemic insulin resistance for which no therapy is currently available. Systems have b...

journal_title：Human gene therapy

authors： Cotugno G,Pollock R,Formisano P,Linher K,Beguinot F,Auricchio A

更新日期：2004-11-01 00:00:00

abstract：:Adeno-associated viral vectors are showing great promise as gene therapy vectors for a wide range of retinal disorders. To date, evaluation of therapeutic approaches has depended almost exclusively on the use of animal models. With recent advances in human stem cell technology, stem cell-derived retina now offers the ...

journal_title：Human gene therapy

authors： Gonzalez-Cordero A,Goh D,Kruczek K,Naeem A,Fernando M,Kleine Holthaus SM,Takaaki M,Blackford SJI,Kloc M,Agundez L,Sampson RD,Borooah S,Ovando-Roche P,Mehat MS,West EL,Smith AJ,Pearson RA,Ali RR

更新日期：2018-10-01 00:00:00

abstract：:Advances in cell and gene therapy are opening up new avenues for regenerative medicine. Because of their acquired pluripotency, human induced pluripotent stem cells (hiPSCs) are a promising source of autologous cells for regenerative medicine. They show unlimited self-renewal while retaining the ability, in principle,...

journal_title：Human gene therapy

authors： Garate Z,Davis BR,Quintana-Bustamante O,Segovia JC

更新日期：2013-06-01 00:00:00

abstract：:The initial stages following the in vitro cytokine stimulation of human cord blood CD34+ cells overlap with the period when lentiviral gene transfer is typically performed. Single-cell transcriptional profiling and time-lapse microscopy were used to investigate how the vector-cell crosstalk impacts on the fate decisio...

journal_title：Human gene therapy

authors： Moussy A,Papili Gao N,Corre G,Poletti V,Majdoul S,Fenard D,Gunawan R,Stockholm D,Páldi A

更新日期：2019-08-01 00:00:00

abstract：:Low in vivo transduction efficiency and safety concerns have been hurdles for effective hematopoietic stem cell (HSC) gene therapy. Here, we investigate whether the safety and efficiency of retroviral gene transfer into HSCs can be improved by using human allogeneic umbilical cord blood (UCB)-derived supplements inste...

journal_title：Human gene therapy

authors： Moon N,Yang SJ,Park BB,Chung YS,Lee JW,Oh IH

更新日期：2008-07-01 00:00:00

abstract：:Engineered measles virus (MV) strains deriving from the vaccine lineage represent a promising oncolytic platform and are currently being tested in phase I trials. In this study, we have demonstrated that MV strains genetically engineered to express the human sodium iodide symporter (NIS) have significant antitumor act...

journal_title：Human gene therapy

authors： Opyrchal M,Allen C,Iankov I,Aderca I,Schroeder M,Sarkaria J,Galanis E

更新日期：2012-04-01 00:00:00

abstract：:Recombinant adeno-associated virus 2 (rAAV2) has been extensively used as a gene delivery vector for the nervous system. It targets primarily neurons in the nervous system and results in sustained long-term expression of transgenes. New rAAV serotypes have been characterized and demonstrated to have improved transduct...

journal_title：Human gene therapy

authors： Burger C,Nash K,Mandel RJ

更新日期：2005-07-01 00:00:00

abstract：:Clinical trials of gene therapy using a viral delivery system for glioma have been limited. Recently, gene therapy using stem cells as the vehicles for delivery of therapeutic agents has emerged as a new treatment strategy for malignant brain tumors. In this study, we used human umbilical cord blood-derived mesenchyma...

journal_title：Human gene therapy

authors： Ryu CH,Park SH,Park SA,Kim SM,Lim JY,Jeong CH,Yoon WS,Oh WI,Sung YC,Jeun SS

更新日期：2011-06-01 00:00:00

abstract：:Clinical gene transfer research has involved adult and child subjects, and it is expected that gene transfer in fetal subjects will occur in the future. Some genetic diseases have serious adverse effects on the fetus before birth, and there is hope that prenatal gene therapy could prevent such disease progression. Res...

journal_title：Human gene therapy

authors： Strong C

更新日期：2011-11-01 00:00:00

abstract：:Protein transduction domains (PTD), which can transport proteins or peptides across biological membranes, have been identified in several proteins of viral, invertebrate, and vertebrate origin. Here, we evaluate the immunological and biological consequences of including PTD in synthetic peptides and in DNA vaccines th...

journal_title：Human gene therapy

authors： Leifert JA,Lindencrona JA,Charo J,Whitton JL

更新日期：2001-10-10 00:00:00

abstract：:Neurodegeneration in Parkinson's disease (PD) affects mainly dopaminergic neurons in the substantia nigra, where age-related, increasing percentages of cells lose detectable respiratory activity associated with depletion of intact mitochondrial DNA (mtDNA). Replenishment of mtDNA might improve neuronal bioenergetic fu...

journal_title：Human gene therapy

authors： Keeney PM,Quigley CK,Dunham LD,Papageorge CM,Iyer S,Thomas RR,Schwarz KM,Trimmer PA,Khan SM,Portell FR,Bergquist KE,Bennett JP Jr

更新日期：2009-08-01 00:00:00

abstract：:Retroviral vectors encoding glucose-responsive promoters driving furin expression may provide an amplified, glucose-regulated secretion of insulin. We constructed LhI*TFSN virus to encode a glucose-regulatable transforming growth factor alpha promoter controlling furin expression with a viral LTR promoter driving cons...

journal_title：Human gene therapy

authors： Barry SC,Ramesh N,Lejnieks D,Simonson WT,Kemper L,Lernmark A,Osborne WR

更新日期：2001-01-20 00:00:00

abstract：:Recombinant adenoviruses have great potential as gene delivery systems because of their ability to infect a wide range of target cells. However, systemic delivery of viral vectors to tissues other than liver and spleen has been inefficient because of the rapid clearance of the circulating virus by the liver. In the pr...

journal_title：Human gene therapy

authors： Ye X,Jerebtsova M,Ray PE

更新日期：2000-03-01 00:00:00

abstract：:The use of synthetic gene delivery systems in human gene transfer is hampered by poor transfection efficiencies, largely because of the inability of DNA to translocate across the nuclear pore complex. A means to overcome this barrier is to bind the DNA to nuclear localization signals (NLSs), which are recognized by sh...

journal_title：Human gene therapy

authors： Mesika A,Kiss V,Brumfeld V,Ghosh G,Reich Z

更新日期：2005-02-01 00:00:00

abstract：:Adeno-associated viral (AAV) vectors are becoming increasingly popular in basic research as well as in clinical gene therapy. Due to its exceptional resistance against physical and chemical stress, however, the increasing use of AAV in laboratories and clinics around the globe raises safety concerns. Proper decontamin...

journal_title：Human gene therapy

authors： Korte J,Mienert J,Hennigs JK,Körbelin J

更新日期：2020-11-06 00:00:00

abstract：:Adeno-associated viral (AAV) vectors containing cone-specific promoters have rescued cone photoreceptor function in mouse and dog models of achromatopsia, but cone-specific promoters have not been optimized for use in primates. Using AAV vectors administered by subretinal injection, we evaluated a series of promoters ...

journal_title：Human gene therapy

authors： Ye GJ,Budzynski E,Sonnentag P,Nork TM,Sheibani N,Gurel Z,Boye SL,Peterson JJ,Boye SE,Hauswirth WW,Chulay JD

更新日期：2016-01-01 00:00:00

abstract：:Despite improvements in drug and device therapy for heart failure, hospitalization rates and mortality have changed little in the past decade. Randomized clinical trials using gene transfer to improve function of the failing heart are the focus of this review. Four randomized clinical trials of gene transfer in heart ...

journal_title：Human gene therapy

authors： Penny WF,Hammond HK

更新日期：2017-05-01 00:00:00

abstract：:Lentiviral vectors are efficiently pseudotyped with RD114-TR, a chimeric envelope glycoprotein made of the extracellular and transmembrane domains of the feline leukemia virus RD114 and the cytoplasmic tail of the murine leukemia virus amphotropic envelope. RD114-TR-pseudotyped vectors may be concentrated by centrifug...

journal_title：Human gene therapy

authors： Di Nunzio F,Piovani B,Cosset FL,Mavilio F,Stornaiuolo A

更新日期：2007-09-01 00:00:00

abstract：:One of the major obstacles to pulmonary-directed gene therapy using adenoviral vectors is the induction of inflammation. We investigated whether the adenoviral particles that constitute the initial inoculum can serve as an inflammatory stimulus, independent of their ability to express genes that they contain. Viral pa...

journal_title：Human gene therapy

authors： McCoy RD,Davidson BL,Roessler BJ,Huffnagle GB,Janich SL,Laing TJ,Simon RH

更新日期：1995-12-01 00:00:00

abstract：:Targeted delivery of intravenously administered genetically altered cells or stem cells is still in an early stage of investigation. We developed a method of delivering iron oxide (ferumoxide)-labeled mesenchymal stem cells (MSCs) to a targeted area in an animal model by applying an external magnet. Rats with or witho...

journal_title：Human gene therapy

authors： Arbab AS,Jordan EK,Wilson LB,Yocum GT,Lewis BK,Frank JA

更新日期：2004-04-01 00:00:00

abstract：:Recent advances in genome sequencing have greatly improved our ability to understand and identify the causes of genetic diseases. However, there remains an urgent need for innovative, safe, and effective treatments for these diseases. CRISPR-based genome editing systems have become important and powerful tools in the ...

journal_title：Human gene therapy

authors： Carlaw TM,Zhang LH,Ross CJD

更新日期：2020-08-01 00:00:00

abstract：:The enzyme hypoxanthine-guanine phosphoribosyltransferase (HGPRT) expressed by the parasite Trypanosoma brucei (Tb) can convert allopurinol, a purine analogue, to corresponding nucleotides with greater efficiency than its human homologue. We have developed a retroviral system that expresses the parasitic enzyme and te...

journal_title：Human gene therapy

authors： Trudeau C,Yuan S,Galipeau J,Benlimame N,Alaoui-Jamali MA,Batist G

更新日期：2001-09-01 00:00:00

abstract：:Human serum is known to inactivate many retroviruses, including murine leukemia viruses (MLV). Exposure of vectors based on MLV to human serum components would presumably decrease the efficiency of gene transfer in vivo. Human serum also lyses xenogeneic cells, which would affect the survival of retroviral vector pack...

journal_title：Human gene therapy

authors： Russell DW,Berger MS,Miller AD

更新日期：1995-05-01 00:00:00

abstract：:Skeletal muscle represents an attractive target tissue for adenoviral gene therapy to treat muscle disorders and as a production platform for systemic expression of therapeutic proteins. However, adenovirus serotype 5 vectors do not efficiently transduce adult muscle tissue. Here we evaluated whether capsid modificati...

journal_title：Human gene therapy

authors： Guse K,Suzuki M,Sule G,Bertin TK,Tyynismaa H,Ahola-Erkkilä S,Palmer D,Suomalainen A,Ng P,Cerullo V,Hemminki A,Lee B

更新日期：2012-10-01 00:00:00

abstract：:Laminin-5 is composed of three distinct polypeptides, alpha3, beta3, and gamma2, which are encoded by three different genes, LAMA3, LAMB3, and LAMC2, respectively. We have isolated epidermal keratinocytes from a patient presenting with a lethal form of junctional epidermolysis bullosa characterized by a homozygous mut...

journal_title：Human gene therapy

authors： Dellambra E,Vailly J,Pellegrini G,Bondanza S,Golisano O,Macchia C,Zambruno G,Meneguzzi G,De Luca M

更新日期：1998-06-10 00:00:00

abstract：:Development of multidrug resistance (MDR) is the major obstacle to successful cancer chemotherapy. We have developed Daudi human lymphoma cells that are 20-fold more resistant than the parent cell line to vincristine (VCR) by infecting cells with pHaMDR1/A retroviral vector (Daudi/MDR20). Three DNA sequences of anti-M...

journal_title：Human gene therapy

authors： Wang FS,Kobayashi H,Liang KW,Holland JF,Ohnuma T

更新日期：1999-05-01 00:00:00

abstract：:A human immunodeficiency virus type 1 (HIV-1)-based retroviral vector pseudotyped with HIV envelope containing the herpes simplex virus-thymidine kinase (HSV-TK) gene under the control of the HIV LTR promoter (pHXTKN) was constructed and stably transferred into human CD4(+) H9, CEM, and U937 cells. RNase protection as...

journal_title：Human gene therapy

authors： Miyake K,Iijima O,Suzuki N,Matsukura M,Shimada T

更新日期：2001-02-10 00:00:00

abstract：:Adenovirus-based vectors comprise the most frequently used vector type in clinical studies to date. Both intense lab research and insights from the clinical trials reveal the importance of a comprehensive understanding of vector-host interactions. Especially for systemic intravenous adenovirus vector delivery, it is p...

journal_title：Human gene therapy

authors： Hagedorn C,Kreppel F

更新日期：2017-10-01 00:00:00

abstract：:Metabolic myopathies are a diverse group of rare diseases in which impaired breakdown of stored energy leads to profound muscle dysfunction ranging from exercise intolerance to severe muscle wasting. Metabolic myopathies are largely caused by functional deficiency of a single gene and are generally subcategorized into...

journal_title：Human gene therapy

authors： Mah CS,Soustek MS,Todd AG,McCall A,Smith BK,Corti M,Falk DJ,Byrne BJ

更新日期：2013-11-01 00:00:00