Corrective transduction of human epidermal stem cells in laminin-5-dependent junctional epidermolysis bullosa.

abstract：:The immune response against human immunodeficiency virus type-1 (HIV-1) is believed to play a role in controlling the early stages of disease progression. The cellular immune response, in particular cytotoxic T lymphocyte (CTL) activity, may be important for eliminating virally infected cells in HIV-1-infected individ...

journal_title：Human gene therapy

authors： Laube LS,Burrascano M,Dejesus CE,Howard BD,Johnson MA,Lee WT,Lynn AE,Peters G,Ronlov GS,Townsend KS

更新日期：1994-07-01 00:00:00

abstract：:Artemis is a hairpin-opening endonuclease involved in nonhomologous end-joining and V(D)J recombination. Deficiency of Artemis results in radiation-sensitive severe combined immunodeficiency (SCID) characterized by complete absence of T and B cells due to an arrest at the receptor recombination stage. We have generate...

journal_title：Human gene therapy

authors： Multhaup M,Karlen AD,Swanson DL,Wilber A,Somia NV,Cowan MJ,McIvor RS

更新日期：2010-07-01 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：:Recombinant adeno-associated virus (rAAV) is a prototypical gene therapy vector characterized by excellent safety profiles, wide host range, and the ability to transduce differentiated cells. Numerous rAAV-based vectors providing efficient and sustained expression of transgenes in target tissues have been developed fo...

journal_title：Human gene therapy

authors： Zolotukhin S

更新日期：2005-05-01 00:00:00

abstract：:Somatic gene therapy using nonautologous recombinant cells immunologically protected with alginate microcapsules has been successfully used to treat rodent genetic diseases. We now report the delivery of recombinant gene products to the brain in rodents by implanting microencapsulated cells for the purpose of eventual...

journal_title：Human gene therapy

authors： Ross CJ,Ralph M,Chang PL

更新日期：1999-01-01 00:00:00

abstract：:Genome engineering has gone mainstream because of breakthroughs in defining and harnessing naturally occurring, customizable DNA recognition cursors (protein or RNA-guided). At present, most gene editing relies on these cursors to direct custom DNA endonucleases to a specific genomic sequence to induce a double-strand...

journal_title：Human gene therapy

authors： Martínez-Gálvez G,Ata H,Campbell JM,Ekker SC

更新日期：2016-06-01 00:00:00

abstract：:Activation of T cells is necessary for efficient retroviral-mediated gene transfer. In addition, if the population of infused cells is to be limited to transduced cells, a means of positive selection is required. We describe a clinical scale procedure for activation of donor T cells with anti-CD3/CD28 beads followed b...

journal_title：Human gene therapy

authors： Orchard PJ,Blazar BR,Burger S,Levine B,Basso L,Nelson DM,Gordon K,McIvor RS,Wagner JE,Miller JS

更新日期：2002-05-20 00:00:00

abstract：:Current clinical gene therapy protocols for the treatment of human immunodeficiency virus type 1 (HIV-1) infection often involve the ex vivo transduction and expansion of CD4+ T cells derived from HIV-positive patients at a late stage in their disease (CD4 count <400). These protocols involve the transduction of T cel...

journal_title：Human gene therapy

authors： Poznansky MC,Foxall R,Mhashilkar A,Coker R,Jones S,Ramstedt U,Marasco W

更新日期：1998-03-01 00:00:00

abstract：:In vitro delivery of interferon-alpha (IFN-alpha) to cultured human monocytes by means of a replication-incompetent herpesvirus vector inhibits human immunodeficiency virus (HIV) replication. To explore the possibility of IFN-alpha gene delivery by vector-infected human monocytes, monocytes were isolated and the cultu...

journal_title：Human gene therapy

authors： Weir JP,Dacquel EJ,Aronovitz J

更新日期：1996-07-10 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：:Lentiviral vectors are promising tools for gene transfer into the central nervous system. We have characterized in detail transduction with human immunodeficiency virus type 1 (HIV-1)-derived vectors encoding enhanced green fluorescent protein (eGFP) in the adult mouse brain. Different brain regions such as the striat...

journal_title：Human gene therapy

authors： Baekelandt V,Claeys A,Eggermont K,Lauwers E,De Strooper B,Nuttin B,Debyser Z

更新日期：2002-05-01 00:00:00

abstract：:Previous studies have documented that the skin can be used as a bioreactor to produce proteins for systemic release to treat diseases. A gene-switch system has been developed that allows regulated expression of therapeutic genes. To determine whether this system could be used in the skin, we developed a transgenic mou...

journal_title：Human gene therapy

authors： Cao T,Tsai SY,O'Malley BW,Wang XJ,Roop DR

更新日期：2002-06-10 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：:Overexpression of human manganese-containing superoxide dismutase (MnSOD) activity has been demonstrated to suppress malignancy in human melanoma and breast carcinoma cells in vitro and in vivo. To study its effects on human oral squamous carcinoma cells, stable transfection and expression of MnSOD in SCC-25 cells hav...

journal_title：Human gene therapy

authors： Liu R,Oberley TD,Oberley LW

更新日期：1997-03-20 00:00:00

abstract：:Gene therapy for heart diseases requires availability of an efficient vector for gene transfer into myocardium. Recombinant adenovirus expressing the Escherichia coli beta-galactosidase (beta-Gal) gene was shown to infect rat cardiocytes efficiently in vivo. However, a time course of gene expression showed that transg...

journal_title：Human gene therapy

authors： Gilgenkrantz H,Duboc D,Juillard V,Couton D,Pavirani A,Guillet JG,Briand P,Kahn A

更新日期：1995-10-01 00:00:00

abstract：:Efficient DNA electrotransfer can be achieved with combinations of short high-voltage (HV) and long low voltage (LV) pulses that cover two effects of the pulses, namely, target cell electropermeabilization and DNA electrophoresis within the tissue. Because HV and LV can be delivered with a lag up to 3000 sec between t...

journal_title：Human gene therapy

authors： Satkauskas S,André F,Bureau MF,Scherman D,Miklavcic D,Mir LM

更新日期：2005-10-01 00:00:00

abstract：:Recombinant adeno-associated virus (AAV) holds much promise for human gene therapy. While evidence indicates that AAV mediates long-term gene transfer in several different tissues, difficulty in preparing and purifying this viral vector in large quantities remains a major obstacle for evaluating AAV vectors in clinica...

journal_title：Human gene therapy

authors： Gao G,Qu G,Burnham MS,Huang J,Chirmule N,Joshi B,Yu QC,Marsh JA,Conceicao CM,Wilson JM

更新日期：2000-10-10 00:00:00

abstract：:The purpose of this study was to determine the safety and antitumor activity of IFN-gamma retroviral vector in patients with advanced melanoma. Seventeen patients (9 single courses, 8 multiple courses) received a total of 363 intratumor injections of IFN-gamma retroviral vector (1 x 10(7) PFU/ml administered at 0.3, 0...

journal_title：Human gene therapy

authors： Nemunaitis J,Fong T,Burrows F,Bruce J,Peters G,Ognoskie N,Meyer W,Wynne D,Kerr R,Pippen J,Oldham F,Ando D

更新日期：1999-05-20 00:00:00

abstract：:Severe fetal growth restriction (FGR) affects 1:500 pregnancies, is untreatable and causes serious neonatal morbidity and death. Reduced uterine blood flow (UBF) and lack of bioavailable VEGF due to placental insufficiency is a major cause. Transduction of uterine arteries in normal or FGR sheep and guinea pigs using ...

journal_title：Human gene therapy

authors： Rossi C,Lees M,Mehta V,Heikura T,Martin J,Zachary I,Spencer R,Peebles DM,Shaw R,Karhinen M,Yla-Herttuala S,David AL

更新日期：2020-11-01 00:00:00

abstract：:Combining chemotherapy and immunotherapy is problematic because chemotherapy can ablate the immune responses initiated by modulators of the immune system. We hypothesized that protection of immunocompetent cells from the toxic effects of chemotherapy, using drug resistance gene therapy strategies, would allow the comb...

journal_title：Human gene therapy

authors： McMillin DW,Hewes B,Gangadharan B,Archer DR,Mittler RS,Spencer HT

更新日期：2006-08-01 00:00:00

abstract：:Glycogen storage disease type II (GSD-II) is a lethal, autosomal recessive metabolic myopathy caused by a lack of acid-alpha-glucosidase (GAA) activity in the cardiac and skeletal muscles. Absence of adequate intralysosomal GAA activity results in massive amounts of glycogen accumulation in multiple muscle groups, res...

journal_title：Human gene therapy

authors： Ding EY,Hodges BL,Hu H,McVie-Wylie AJ,Serra D,Migone FK,Pressley D,Chen YT,Amalfitano A

更新日期：2001-05-20 00:00:00

abstract：:DNA expression vectors may be administered to patients like conventional medicines to have a finite and controlled duration of action. The clinical application of these medicines will require a precise understanding of the kinetics of the administered gene, the mRNA transcript, and the gene product. The apparent kinet...

journal_title：Human gene therapy

authors： Ledley TS,Ledley FD

更新日期：1994-06-01 00:00:00

abstract：:Retinal gene therapy based on adeno-associated viral (AAV) vectors is safe and efficient in humans. The low intrinsic DNA transfer capacity of AAV has been expanded by dual vectors where a large expression cassette is split in two halves independently packaged in two AAV vectors. Dual AAV transduction efficiency, howe...

journal_title：Human gene therapy

authors： Maddalena A,Dell'Aquila F,Giovannelli P,Tiberi P,Wanderlingh LG,Montefusco S,Tornabene P,Iodice C,Visconte F,Carissimo A,Medina DL,Castoria G,Auricchio A

更新日期：2018-08-01 00:00:00

abstract：:When transferring the human multidrug resistance 1 (MDR1) cDNA, FMEV retroviral vectors mediate high-dose multidrug resistance and, thus, background-free selection in primary human hematopoietic progenitor cells. Here, we analyzed strategies for co-expression of a second gene from an FMEV:MDR1 vector. When linking the...

journal_title：Human gene therapy

authors： Hildinger M,Fehse B,Hegewisch-Becker S,John J,Rafferty JR,Ostertag W,Baum C

更新日期：1998-01-01 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：:Replication-deficient adenoviruses are known to induce acute injury and inflammation of infected tissues, thus limiting their use for human gene therapy. However, molecular mechanisms triggering this response have not been fully defined. To characterize this response, chemokine expression was evaluated in DBA/2 mice f...

journal_title：Human gene therapy

authors： Muruve DA,Barnes MJ,Stillman IE,Libermann TA

更新日期：1999-04-10 00:00:00

abstract：:Virotherapy is a unique modality for the treatment of cancer with oncolytic viruses (OVs) that selectively infect and lyse tumor cells, spread within tumors, and activate anti-tumor immunity. Various viruses are being developed as OVs preclinically and clinically, several of them engineered to encode therapeutic prote...

journal_title：Human gene therapy

authors： Ungerechts G,Engeland CE,Buchholz CJ,Eberle J,Fechner H,Geletneky K,Holm PS,Kreppel F,Kühnel F,Lang KS,Leber MF,Marchini A,Moehler M,Mühlebach MD,Rommelaere J,Springfeld C,Lauer UM,Nettelbeck DM

更新日期：2017-10-01 00:00:00

abstract：:Recent marketing approval for genetically engineered hematopoietic stem and T cells bears witness to the substantial improvements in lentiviral vectors over the last two decades, but evaluations of the long-term efficacy and toxicity of gene and cell therapy products will, nevertheless, require further studies in nonh...

journal_title：Human gene therapy

authors： Sii-Felice K,Castillo Padilla J,Relouzat F,Cheuzeville J,Tantawet S,Maouche L,Le Grand R,Leboulch P,Payen E

更新日期：2019-10-01 00:00:00

abstract：:DNA vaccination is an attractive approach for tumor immunotherapy because of its stability and simplicity of delivery. Advances demonstrate that helper T cell responses play a critical role in initiating immune responses. The aim of the current study is to test whether targeting HPV-16 E7 to the endosomal/lysosomal co...

journal_title：Human gene therapy

authors： Ji H,Wang TL,Chen CH,Pai SI,Hung CF,Lin KY,Kurman RJ,Pardoll DM,Wu TC

更新日期：1999-11-20 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