Exogenous surfactant enhances the delivery of recombinant adenoviral vectors to the lung.

abstract：:A novel retroviral vector has been designed based on a Friend-murine leukemia virus (Fr-MuLV) FB29 strain. The latter has been selected according to characteristics of pathogenicity in mice where it induces a disease of the haemopoietic system affecting all lineages. Higher infectivity has also been demonstrated as co...

journal_title：Human gene therapy

authors： Cohen-Haguenauer O,Restrepo LM,Masset M,Bayer J,Dal Cortivo L,Marolleau JP,Benbunan M,Boiron M,Marty M

更新日期：1998-01-20 00:00:00

abstract：:Gene-modified lymphocytes have a potential role in the therapy of cancer, infectious diseases, and genetic disorders of the immune system. Current gene therapy protocols involving gene transfer into lymphocytes utilize retroviruses with amphotropic envelope proteins. However, transduction efficiencies in lymphocytes u...

journal_title：Human gene therapy

authors： Lam JS,Reeves ME,Cowherd R,Rosenberg SA,Hwu P

更新日期：1996-08-01 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：:Hyperoxia and ischemia-reperfusion cause profound lung cellular damage mediated, in part, by generation of oxygen radicals. We hypothesized that gene therapy can be used to overcome oxidant injury by augmenting intracellular antioxidant enzymes. Adult rats were injected intratracheally with an adenovirus (Ad) vector e...

journal_title：Human gene therapy

authors： Danel C,Erzurum SC,Prayssac P,Eissa NT,Crystal RG,Hervé P,Baudet B,Mazmanian M,Lemarchand P

更新日期：1998-07-01 00:00:00

abstract：:Hemophilia arthropathy (HA) represents the majority of morbidity in severe hemophilia patients, especially in resource-limited countries. Adeno-associated virus (AAV)-mediated gene therapy is showing promise for managing hemophilia. However, patients with neutralizing antibodies (NAbs) against AAV, and inhibitors to c...

journal_title：Human gene therapy

authors： Zhang F,Yan X,Li M,Hua B,Xiao X,Monahan PE,Sun J

更新日期：2020-04-01 00:00:00

abstract：:Mucopolysaccharidosis type II (MPS II) is a neuropathic lysosomal storage disorder caused by a deficiency of iduronate-2-sulfatase (IDS), which leads to the accumulation of glycosaminoglycans (GAGs). We demonstrated that biochemical alterations in the brains of MPS II mice are not corrected by bone marrow transplantat...

journal_title：Human gene therapy

authors： Wakabayashi T,Shimada Y,Akiyama K,Higuchi T,Fukuda T,Kobayashi H,Eto Y,Ida H,Ohashi T

更新日期：2015-06-01 00:00:00

abstract：:Nonviral jet injection is an applicable technology for in vivo gene transfer of naked DNA. However, little is known about the biodistribution and clearance of jet-injected DNA, or about its localization within tissue and cells. Therefore, in this study we analyzed the intratumoral and systemic biodistribution of jet-i...

journal_title：Human gene therapy

authors： Walther W,Minow T,Martin R,Fichtner I,Schlag PM,Stein U

更新日期：2006-06-01 00:00:00

abstract：:Trans-dominant mutants of human immunodeficiency virus type 1 (HIV-1) Tat and Rev are attractive candidates for use in gene therapy in the treatment of HIV-1 infections because both are essential for viral replication. Retroviral vectors were constructed to allow either Tat-inducible or Tat- and Rev-inducible expressi...

journal_title：Human gene therapy

authors： Liem SE,Ramezani A,Li X,Joshi S

更新日期：1993-10-01 00:00:00

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：: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：:Glaucoma, a group of optic neuropathies, is the leading cause of irreversible blindness. Neuronal apoptosis in glaucoma is primarily associated with high intraocular pressure caused by chronically impaired outflow of aqueous humor through the trabecular meshwork, a reticulum of mitotically inactive endothelial-like ce...

journal_title：Human gene therapy

authors： Loewen N,Fautsch MP,Peretz M,Bahler CK,Cameron JD,Johnson DH,Poeschla EM

更新日期：2001-11-20 00:00:00

abstract：:Therapeutic levels of expression of the beta-globin gene have been difficult to achieve with conventional retroviral vectors without the inclusion of DNase I-hypersensitive site (HS2, HS3, and HS4) enhancer elements. We generated recombinant adeno-associated viral (AAV) vectors carrying an antisickling human beta-glob...

journal_title：Human gene therapy

authors： Maina N,Zhong L,Li X,Zhao W,Han Z,Bischof D,Aslanidi G,Zolotukhin S,Weigel-Van Aken KA,Rivers AE,Slayton WB,Yoder MC,Srivastava A

更新日期：2008-04-01 00:00:00

abstract：:Adenoviruses are attractive vectors for gene transfer into cardiac muscle. However, their promiscuous tissue tropism, which leads to an ectopic expression of the transgene, is a considerable limitation. To restrict expression to cardiomyocytes, we have constructed two recombinant adenoviruses (Ad-MLC2-250betagal and A...

journal_title：Human gene therapy

authors： Griscelli F,Gilardi-Hebenstreit P,Hanania N,Franz WM,Opolon P,Perricaudet M,Ragot T

更新日期：1998-09-01 00:00:00

abstract：:Mutations in the alpha-chain of lysosomal hexosaminidase (EC 3.2.1.52) underlie two distinct biochemical phenotypes known as variant B and variant B1 of G(M2) gangliosidosis. This paper shows that the transduction of human B1-type fibroblasts (producing catalytically inactive alpha-chains) with a retroviral vector enc...

journal_title：Human gene therapy

authors： Teixeira CA,Sena-Esteves M,Lopes L,Sá Miranda MC,Ribeiro MG

更新日期：2001-09-20 00:00:00

abstract：:The liver is an attractive target tissue for gene therapy. Current approaches for hepatic gene delivery include retroviral and adenoviral vectors, liposome/DNA, and peptide/DNA complexes. This study describes a technique for direct injection of DNA into liver that led to significant gene expression. Gene expression wa...

journal_title：Human gene therapy

authors： Hickman MA,Malone RW,Lehmann-Bruinsma K,Sih TR,Knoell D,Szoka FC,Walzem R,Carlson DM,Powell JS

更新日期：1994-12-01 00:00:00

abstract：:Gene therapy studies in primates can provide important information regarding vector tropism, specific cellular expression, biodistribution, and safety prior to clinical trials. In this study, we report the assessment of transduction efficiency of recombinant adeno-associated virus (rAAV) vectors using human postmortem...

journal_title：Human gene therapy

authors： Fradot M,Busskamp V,Forster V,Cronin T,Léveillard T,Bennett J,Sahel JA,Roska B,Picaud S

更新日期：2011-05-01 00:00:00

abstract：:Gene electrotransfer is gaining momentum as an efficient methodology for nonviral gene transfer. In skeletal muscle, data suggest that electric pulses play two roles: structurally permeabilizing the muscle fibers and electrophoretically supporting the migration of DNA toward or across the permeabilized membrane. To in...

journal_title：Human gene therapy

authors： André FM,Gehl J,Sersa G,Préat V,Hojman P,Eriksen J,Golzio M,Cemazar M,Pavselj N,Rols MP,Miklavcic D,Neumann E,Teissié J,Mir LM

更新日期：2008-11-01 00:00:00

abstract：:Due to both the avascularity of the cornea and the relatively immune-privileged status of the eye, corneal transplantation is one of the most successful clinical transplant procedures. However, in high-risk patients, which account for >20% of the 180,000 transplants carried out worldwide each year, the rejection rate ...

journal_title：Human gene therapy

authors： Fouladi N,Parker M,Kennedy V,Binley K,McCloskey L,Loader J,Kelleher M,Mitrophanous KA,Stout JT,Ellis S

更新日期：2018-06-01 00:00:00

abstract：:The transfer of a drug resistance gene into hematopoietic cells is an approach being investigated to overcome the problem of myelosuppression produced by anticancer drugs. Chemotherapeutic agents are often given in combination in order to increase their effectiveness. Consequently, there is an advantage in designing v...

journal_title：Human gene therapy

authors： Beauséjour CM,Le NL,Létourneau S,Cournoyer D,Momparler RL

更新日期：1998-11-20 00:00:00

abstract：:Here we show potent inhibition of HIV-1 replication in a human T cell line and primary human CD4(+) cells by expressing a single antiviral protein. Nullbasic is a mutant form of the HIV-1 Tat protein that was previously shown to strongly inhibit HIV-1 replication in nonhematopoietic cell lines by targeting three steps...

journal_title：Human gene therapy

authors： Apolloni A,Lin MH,Sivakumaran H,Li D,Kershaw MH,Harrich D

更新日期：2013-03-01 00:00:00

abstract：:Mitochondrial neurogastrointestinal encephalomyopathy (MNGIE) is a devastating disease caused by mutations in TYMP, which encodes thymidine phosphorylase (TP). In MNGIE patients, TP dysfunction results in systemic thymidine and deoxyuridine overload, which interferes with mitochondrial DNA replication. Preclinical stu...

journal_title：Human gene therapy

authors： Cabrera-Pérez R,Vila-Julià F,Hirano M,Mingozzi F,Torres-Torronteras J,Martí R

更新日期：2019-08-01 00:00:00

abstract：:pZIG(hGCSFR) is a retroviral vector that can co-express two genes and also provides alternative selection markers. This retroviral vector has been constructed to incorporate an internal ribosome entry site (IRES) element to co-express two exogenous genes in mammalian cells. Two marker/selection genes have been cloned ...

journal_title：Human gene therapy

authors： Saleh M

更新日期：1997-05-20 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：:The use of viral thymidine kinase (TK) gene coupled with the administration of ganciclovir to render cancer cell death has been studied extensively. Many of these experiments utilized retrovirus to transfer the TK gene under the control of a nonspecific promoter. Because nonspecific expression of the viral TK gene may...

journal_title：Human gene therapy

authors： Su H,Chang JC,Xu SM,Kan YW

更新日期：1996-03-01 00:00:00

abstract：:In the next decades, gene editing technologies are expected to be used in the treatment and prevention of human diseases. Yet, the future uses of gene editing in medicine are still unknown, including its applicability and effectiveness to the treatment and prevention of infectious diseases, cancer, and monogenic and p...

journal_title：Human gene therapy

authors： Rocha LFM,Braga LAM,Mota FB

更新日期：2020-08-01 00:00:00

abstract：:Three-vessel disease (TVD) is a severe coronary heart disease (CHD) with poor prognosis. Niemann-Pick C1-like 1 (NPC1L1) is a transporter protein for exogenous cholesterol absorption, and 3-hydroxy-3-methylglutaryl-coenzyme A reductase (HMGCR) is a rate-limiting enzyme for cholesterol synthesis. We aimed to investigat...

journal_title：Human gene therapy

authors： Zhao X,Li J,Tang X,Liu R,Xu J,Xu L,Jiang L,Huang K,Tian J,Feng X,Wu Y,Zhang Y,Wang D,Sun K,Xu B,Zhao W,Hui R,Gao R,Song L,Yuan J

更新日期：2021-01-22 00:00:00

abstract：:Recombinant vectors based on adeno-associated virus serotype 8 (AAV8) have been successfully used in the clinic and hold great promise for liver-directed gene therapy. Preexisting immunity against AAV8 or the development of antibodies against the therapeutic transgene product might negatively affect the outcomes of ge...

journal_title：Human gene therapy

authors： Ferla R,Claudiani P,Savarese M,Kozarsky K,Parini R,Scarpa M,Donati MA,Sorge G,Hopwood JJ,Parenti G,Fecarotta S,Nigro V,Sivri HS,Van Der Ploeg A,Andria G,Brunetti-Pierri N,Auricchio A

更新日期：2015-03-01 00:00:00

abstract：:Interleukin-12 (IL-12) is a heterodimeric cytokine that plays an important role in the development of cellular immunity. Clinical applications for this lymphokine include resolution of infectious disease, cancer immunotherapy, and boosting cellular immunity in AIDS patients. When using IL-12 and other cytokines therap...

journal_title：Human gene therapy

authors： Bramson J,Hitt M,Gallichan WS,Rosenthal KL,Gauldie J,Graham FL

更新日期：1996-02-10 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：:Recombinant adeno-associated virus serotype 2 (rAAV2)-based human gene therapy for cystic fibrosis has progressed through a series of preclinical studies and phase I and II clinical trials. This agent has shown an encouraging safety profile, consistent levels of DNA transfer, and positive evidence of short-term clinic...

journal_title：Human gene therapy

authors： Flotte TR,Schwiebert EM,Zeitlin PL,Carter BJ,Guggino WB

更新日期：2005-08-01 00:00:00