Recombinant adeno-associated viral vectors in the nervous system.

abstract：:The large amounts of recombinant adeno-associated virus (rAAV) vector needed for clinical trials and eventual commercialization require robust, economical, reproducible, and scalable production processes compatible with current good manufacturing practice. rAAV produced using baculovirus and insect cells satisfies the...

journal_title：Human gene therapy

authors： Cecchini S,Virag T,Kotin RM

更新日期：2011-08-01 00:00:00

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：:Replication-deficient adenovirus vector (Ad) is one of the most efficient gene transfer vehicles for human gene therapy. However, Ad is antigenic, known to evoke prominent inflammatory responses in vivo, and there are concerns that using Ad in patients with immune-mediated disorders (allergy and autoimmune diseases) m...

journal_title：Human gene therapy

authors： Suzuki M,Suzuki S,Yamamoto N,Komatsu S,Inoue S,Hashiba T,Nishikawa M,Ishigatsubo Y

更新日期：2000-04-10 00:00:00

abstract：:Genetically modified lymphoblastoid cell lines (LCL) have been shown to be an attractive alternative source of antigen-presenting cells for cancer vaccination in vitro. We tested their application in patients with pancreatic cancer in a phase I clinical trial. As a model tumor antigen, we selected the point-mutated (c...

journal_title：Human gene therapy

authors： Kubuschok B,Pfreundschuh M,Breit R,Hartmann F,Sester M,Gärtner B,König J,Murawski N,Held G,Zwick C,Neumann F

更新日期：2012-12-01 00:00:00

abstract：:Rat myoblast primary cultures were tested as a model for proinsulin synthesis and processing and unregulated insulin delivery for insulin-dependent diabetes mellitus (IDDM) gene therapy. Three human proinsulin cDNA constructs containing genetically engineered furin endoprotease cleavage sites between the B-chain and C...

journal_title：Human gene therapy

authors： Simonson GD,Groskreutz DJ,Gorman CM,MacDonald MJ

更新日期：1996-01-01 00:00:00

abstract：:Engineering gene therapy vectors to modulate the immune response is an important goal. In this regard, costimulation of T cells is a critical determinant in immune activation. The costimulatory molecule CD40, expressed on antigen-presenting cells, is thought to interact with CD40 ligand (CD40L) expressed on activated ...

journal_title：Human gene therapy

authors： Sin JI,Kim JJ,Zhang D,Weiner DB

更新日期：2001-06-10 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：:Ornithine transcarbamylase deficiency (OTCD) is an inborn error of urea synthesis that has been considered as a model for liver-directed gene therapy. Current treatment has failed to avert a high mortality or morbidity from hyperammonemic coma. Restoration of enzyme activity in the liver should suffice to normalize me...

journal_title：Human gene therapy

authors： Raper SE,Yudkoff M,Chirmule N,Gao GP,Nunes F,Haskal ZJ,Furth EE,Propert KJ,Robinson MB,Magosin S,Simoes H,Speicher L,Hughes J,Tazelaar J,Wivel NA,Wilson JM,Batshaw ML

更新日期：2002-01-01 00:00:00

abstract：:Gutted adenoviral (Ad) vectors have a greater cloning capacity and elicit less immune response than conventional Ad vectors. Unfortunately, clinical use of gutted vectors has been slowed by production difficulties, including low yield and a tendency for recombinant virus to emerge. These two problems are related, beca...

journal_title：Human gene therapy

authors： Hartigan-O'Connor D,Barjot C,Crawford R,Chamberlain JS

更新日期：2002-03-01 00:00:00

abstract：:Transfer of a herpes simplex virus-derived thymidine kinase (HSV-tk) gene into brain tumor cells and subsequent ganciclovir (GCV) treatment has been shown by others to be an effective treatment in rats with intracerebrally inoculated 9L gliosarcomas. Mechanism of action and reproducibility are, however, still a matter...

journal_title：Human gene therapy

authors： Vincent AJ,Vogels R,Someren GV,Esandi MC,Noteboom JL,Avezaat CJ,Vecht C,Bekkum DW,Valerio D,Bout A,Hoogerbrugge PM

更新日期：1996-01-20 00:00:00

abstract：:Defined serum-free conditions have great conceptual advantages for the biological safety and standardization of clinical gene transfer into hematopoietic stem cells. In the only study reported to date, Sekhar et al. achieved low serum conditions by a complex concentration procedure of a retroviral supernatant initiall...

journal_title：Human gene therapy

authors： Glimm H,Flügge K,Möbest D,Hofmann VM,Postmus J,Henschler R,Lange W,Finke J,Kiem HP,Schulz G,Rosenthal F,Mertelsmann R,von Kalle C

更新日期：1998-04-10 00:00:00

abstract：:The epithelial-mesenchymal transition (EMT) has been recognized to occur during embryonic development, fibrosis, and tumor metastasis. Nuclear factor (NF)-κB plays a central role in mediating the inflammation and wound-healing responses during liver fibrogenesis. However, the involvement of NF-κB during EMT in liver c...

journal_title：Human gene therapy

authors： Kim KH,Lee WR,Kang YN,Chang YC,Park KK

更新日期：2014-08-01 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：:The addition of replication-defective recombinant adenovirus to plasmid transfection (termed here "adenofection") has been shown to increase plasmid transgene expression in limited studies. Similarly, the addition of cationic liposomes to adenovirus increases adenovirus-mediated gene transduction (termed here "lipoduc...

journal_title：Human gene therapy

authors： Dunphy EJ,Redman RA,Herweijer H,Cripe TP

更新日期：1999-09-20 00:00:00

abstract：:Classical gene therapy for cystic fibrosis has had limited success because of immune response against viral vectors and short-term expression of cDNA-based transgenes. These limitations could be overcome by delivering the complete genomic CFTR gene on nonintegrating human artificial chromosomes (HACs). Here, we report...

journal_title：Human gene therapy

authors： Rocchi L,Braz C,Cattani S,Ramalho A,Christan S,Edlinger M,Ascenzioni F,Laner A,Kraner S,Amaral M,Schindelhauer D

更新日期：2010-09-01 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：: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：:At present there is no known effective pharmacological therapy for acute lung injury (ALI). Because keratinocyte growth factor (KGF) promotes epithelial cell growth, intratracheal administration of KGF has the possibility of restoring lung tissue integrity in injured lungs and improving patient outcomes. However, trea...

journal_title：Human gene therapy

authors： Baba Y,Yazawa T,Kanegae Y,Sakamoto S,Saito I,Morimura N,Goto T,Yamada Y,Kurahashi K

更新日期：2007-02-01 00:00:00

abstract：:CRISPR-based technology has been adapted to achieve a wide range of genome modifications including transcription regulation. The focus of this review is on the application of CRISPR-based platforms such as nuclease-deficient Cas9 and Cas12a, to achieve targeted gene activation. We review studies to date that have empl...

journal_title：Human gene therapy

authors： Lek A,Ma K,Woodman K,Lek M

更新日期：2021-01-15 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：:Efficient and homogeneous gene transfer to cardiac myocytes is a major target in myocardial gene therapy. The aim of this study was to determine the conditions permitting efficient, homogeneous, adenovirus-mediated gene transfer to cardiac myocytes, with a view to application during coronary artery catheterization. Ge...

journal_title：Human gene therapy

authors： Logeart D,Hatem SN,Rücker-Martin C,Chossat N,Névo N,Haddada H,Heimburger M,Perricaudet M,Mercadier JJ

更新日期：2000-05-01 00:00:00

abstract：:Bag-1 exerts powerful antiapoptotic effects by binding and stabilizing Bcl-2 and interacting with the tumor necrosis factor receptor type I-induced death signal. We examined the effects of overexpression of Bag-1 by ex vivo adenoviral gene transfer on cold (4 degrees C for 24 hr) ischemia/reperfusion (I/R) injury of r...

journal_title：Human gene therapy

authors： Sawitzki B,Amersi F,Ritter T,Fisser M,Shen XD,Ke B,Busuttil R,Volk HD,Kupiec-Weglinski JW

更新日期：2002-08-10 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：:Human hematopoietic stem cells (HSCs) are poorly transduced by vectors based on adenovirus serotype 5 (Ad5). This is primarily due to the paucity of the coxsackievirus-Ad receptor on these cells. In an attempt to change the tropism of Ad5, we constructed a series of chimeric E1-deleted Ad5 vectors in which the shaft a...

journal_title：Human gene therapy

authors： Knaän-Shanzer S,Van Der Velde I,Havenga MJ,Lemckert AA,De Vries AA,Valerio D

更新日期：2001-11-01 00:00:00

abstract：:C-C chemokine receptor type 5 (CCR5) is a major co-receptor for the entry of human immunodeficiency virus type-1 (HIV-1) into target cells. Human hematopoietic stem cells (hHSCs) with naturally occurring CCR5 deletions (Δ32) or artificially disrupted CCR5 have shown potential for curing acquired immunodeficiency syndr...

journal_title：Human gene therapy

authors： Yao Y,Nashun B,Zhou T,Qin L,Qin L,Zhao S,Xu J,Esteban MA,Chen X

更新日期：2012-02-01 00:00:00

abstract：:Successful gene transfer into articular cartilage is a prerequisite for gene therapy of articular joint disorders. In the present study we tested the hypothesis that recombinant adeno-associated virus (rAAV) vectors are capable of effecting gene transfer in isolated articular chondrocytes in vitro, articular cartilage...

journal_title：Human gene therapy

authors： Madry H,Cucchiarini M,Terwilliger EF,Trippel SB

更新日期：2003-03-01 00:00:00

abstract：:Human adenoviruses are the most widely used vectors in gene medicine, with applications ranging from oncolytic therapies to vaccinations, but adenovirus vectors are not without side effects. In addition, natural adenoviruses pose severe risks for immunocompromised people, yet infections are usually mild and self-limit...

journal_title：Human gene therapy

authors： Hendrickx R,Stichling N,Koelen J,Kuryk L,Lipiec A,Greber UF

更新日期：2014-04-01 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：:Adenovirus-polylysine-DNA complexes were evaluated for their capacity to accomplish direct in vivo gene transfer to airway epithelium employing a rodent model. Binary complexes containing transferrin or adenovirus, or combination complexes containing both transferrin and adenovirus, were evaluated. The highest in vitr...

journal_title：Human gene therapy

authors： Gao L,Wagner E,Cotten M,Agarwal S,Harris C,Rømer M,Miller L,Hu PC,Curiel D

更新日期：1993-02-01 00:00:00

abstract：:The therapeutic use of neurotrophic factors for neurodegenerative diseases is promising, however, optimal methods for continuous delivery of these substances to the human central nervous system (CNS) remains problematic. One approach would be to graft genetically engineered human cells that continuously secrete high l...

journal_title：Human gene therapy

authors： Lin Q,Cunningham LA,Epstein LG,Pechan PA,Short MP,Fleet C,Bohn MC

更新日期：1997-02-10 00:00:00