Targeted gene correction in the mdx mouse using short DNA fragments: towards application with bone marrow-derived cells for autologous remodeling of dystrophic muscle.

abstract：:Platelet-derived growth factor-BB (PDGF-BB) stimulates repair of healing-impaired chronic wounds such as diabetic ulcers and periodontal lesions. However, limitations in predictability of tissue regeneration occur due, in part, to transient growth factor bioavailability in vivo. Here, we report that gene delivery of P...

journal_title：Gene therapy

authors： Chang PC,Seol YJ,Cirelli JA,Pellegrini G,Jin Q,Franco LM,Goldstein SA,Chandler LA,Sosnowski B,Giannobile WV

更新日期：2010-01-01 00:00:00

abstract：:Self-amplifying RNA (saRNA) is a promising biotherapeutic tool that has been used as a vaccine against both infectious diseases and cancer. saRNA has been shown to induce protein expression for up to 60 days and elicit immune responses with lower dosing than messenger RNA (mRNA). Because saRNA is a large (~9500 nt), n...

journal_title：Gene therapy

authors： Blakney AK,McKay PF,Yus BI,Aldon Y,Shattock RJ

更新日期：2019-09-01 00:00:00

abstract：:The concept and application of ethical principles in the context of medical research is changing rapidly. This is especially true for fast moving fields such as gene therapy, a relatively new but rapidly maturing field offering opportunities to influence health at a fundamental level, which may become a cornerstone of...

journal_title：Gene therapy

authors： Spink J,Geddes D

更新日期：2004-11-01 00:00:00

abstract：:Cationic lipid-DNA complexes (lipoplexes) have been widely used as gene transfer vectors which avoid the adverse immunogenicity and potential for viraemia of viral vectors. With the long-term aim of gene transfer into skeletal muscle in vivo, we describe a direct in vitro comparison of two commercially available catio...

journal_title：Gene therapy

authors： Dodds E,Dunckley MG,Naujoks K,Michaelis U,Dickson G

更新日期：1998-04-01 00:00:00

abstract：:Because periodontal ligament (PDL) cells are reported to contain progenitor or stem cell populations, they are considered a beneficial cell source for clinical periodontal regeneration. Both bone morphogenetic protein 4 (BMP4) and human telomerase reverse transcriptase (hTERT) have essential roles in the modulation of...

journal_title：Gene therapy

authors： Mi HW,Lee MC,Fu E,Chow LP,Lin CP

更新日期：2011-05-01 00:00:00

abstract：:We demonstrated previously that the additive-type recombinant Sendai virus (rSeV) is highly efficient for use in pulmonary gene transfer; however, rSeV exhibits inflammatory responses. To overcome this problem, we tested newly developed non-transmissible constructs, namely, temperature-sensitive F-deleted vector, rSeV...

journal_title：Gene therapy

authors： Tanaka S,Yonemitsu Y,Yoshida K,Okano S,Kondo H,Inoue M,Hasegawa M,Masumoto K,Suita S,Taguchi T,Sueishi K

更新日期：2007-07-01 00:00:00

abstract：:Gene therapy of cystic fibrosis (CF) lung disease needs highly efficient delivery and long-lasting complementation of the CFTR (cystic fibrosis transmembrane conductance regulator) gene into the respiratory epithelium. The development of lentiviral vectors has been a recent advance in the field of gene transfer and th...

journal_title：Gene therapy

authors： Copreni E,Penzo M,Carrabino S,Conese M

更新日期：2004-10-01 00:00:00

abstract：:The use of adenoviral vectors as potent gene delivery systems requires expression of the Coxsackievirus/adenovirus receptor (CVADR) on the target cell surface. This receptor is important for virus attachment to the cell surface. For effective internalization of the vector into the target cell the integrins alpha(v)bet...

journal_title：Gene therapy

authors： Loskog A,Hedlund T,Wester K,de la Torre M,Philipson L,Malmström PU,Tötterman TH

更新日期：2002-05-01 00:00:00

abstract：:Radiosensitization of human gastrointestinal tumors by 5-fluorouracil (5-FU) has been studied in vitro and clinically in human cancer therapy trials. The bacterial enzyme cytosine deaminase (CD) converts the nontoxic prodrug 5-fluorocytosine (5-FC) into 5-FU. Human colon cancer cells stably expressing CD have been sho...

journal_title：Gene therapy

authors： Stackhouse MA,Pederson LC,Grizzle WE,Curiel DT,Gebert J,Haack K,Vickers SM,Mayo MS,Buchsbaum DJ

更新日期：2000-06-01 00:00:00

abstract：:Alveolar bone loss associated with periodontal diseases is the result of osteoclastogenesis induced by bacterial pathogens. The mitogen-activated protein kinase (MAPK) phosphatase 1 (MKP-1) is a critical negative regulator of immune response as a key phosphatase capable of dephosphorylating activated MAPKs. In this st...

journal_title：Gene therapy

authors： Yu H,Li Q,Herbert B,Zinna R,Martin K,Junior CR,Kirkwood KL

更新日期：2011-04-01 00:00:00

abstract：:Gene transfer technology has opened novel treatment avenues toward the treatment of damaged musculoskeletal tissues, and may be particularly beneficial to articular cartilage. There is no natural repair mechanism to heal damaged or diseased cartilage. Existing pharmacologic, surgical and cell based treatments may offe...

journal_title：Gene therapy

authors： Trippel SB,Ghivizzani SC,Nixon AJ

更新日期：2004-02-01 00:00:00

abstract：:Aptamers are oligonucleotides evolved in vitro or in nature to bind target ligands with high affinity and specificity. They are emerging as powerful tools in the fields of therapeutics, drug development, target validation and diagnostics. Aptamers are attractive alternatives to antibody- and small-molecule-based thera...

journal_title：Gene therapy

authors： Que-Gewirth NS,Sullenger BA

更新日期：2007-02-01 00:00:00

abstract：:The accessibility of adipose tissue and its ability to secrete various bioactive molecules suggest that adipose cells may be attractive targets for gene therapy applications. Here, we report the use of highly defective herpes simplex virus (HSV) vectors as suitable gene transfer agents for adipose cells in culture and...

journal_title：Gene therapy

authors： Fradette J,Wolfe D,Goins WF,Huang S,Flanigan RM,Glorioso JC

更新日期：2005-01-01 00:00:00

abstract：:Mutations in GJB2, which codes for the gap junction (GJ) protein connexin26 (Cx26), are the most common causes of human nonsyndromic hereditary deafness. We inoculated modified adeno-associated viral (AAV) vectors into the scala media of early postnatal conditional Gjb2 knockout mice to drive exogenous Cx26 expression...

journal_title：Gene therapy

authors： Yu Q,Wang Y,Chang Q,Wang J,Gong S,Li H,Lin X

更新日期：2014-01-01 00:00:00

abstract：:Recombinant Semliki Forest virus (SFV) is an attractive viral vector system owing to its ability to allow high efficiency of viral protein expression. To produce recombinant pseudotyped human immunodeficiency virus type 1 (HIV-1) virions, we designed a chimeric SFV/HIV vector system that contains both the HIV-1 cis- a...

journal_title：Gene therapy

authors： Del Vecchio C,Calistri A,Lombardi G,Celegato M,Biasolo MA,Palù G,Parolin C

更新日期：2009-02-01 00:00:00

abstract：:A major concern of using viral gene therapy is the potential for uncontrolled vector propagation and infection that might result in serious deleterious effects. To enhance the safety, several viral vectors, including vectors based on Sindbis virus, were engineered to lose their capability to replicate and spread after...

journal_title：Gene therapy

authors： Tseng JC,Daniels G,Meruelo D

更新日期：2009-02-01 00:00:00

abstract：:Transplantation faces several major obstacles that could be overcome by expression of immunomodulatory proteins through application of gene therapy techniques. Gene therapy strategies to prolong graft survival involve gene transfer of immunosuppressive or graft-protecting molecules. Very promising results have been ob...

journal_title：Gene therapy

authors： Guillot C,Le Mauff B,Cuturi MC,Anegon I

更新日期：2000-01-01 00:00:00

abstract：:Prenatal gene therapy has been considered for Herlitz junctional epidermolysis bullosa (H-JEB), a lethal genodermatosis caused by the absence of any of the three subunits of laminin-5, resulting from birth in widespread blistering and erosions of skin and mucosae. To investigate this strategy in an animal model, adeno...

journal_title：Gene therapy

authors： Mühle C,Neuner A,Park J,Pacho F,Jiang Q,Waddington SN,Schneider H

更新日期：2006-12-01 00:00:00

abstract：:The existence of cells with stem cell-like abilities derived from various tissues can now be extended to include the skeletal muscle compartment. Although researchers have focused on the utilization of these cells with regard to their myogenic capacity, initially exploring more efficient cellular therapy treatments fo...

journal_title：Gene therapy

authors： Jankowski RJ,Deasy BM,Huard J

更新日期：2002-05-01 00:00:00

abstract：:We investigated the efficiency of activated polyamidoamine dendrimers, a new class of nonviral vectors, to transfect rabbit and human corneas in ex vivo culture. In addition to assessing the expression of a marker gene we have demonstrated that this approach can be used to induce the production of TNF receptor fusion ...

journal_title：Gene therapy

authors： Hudde T,Rayner SA,Comer RM,Weber M,Isaacs JD,Waldmann H,Larkin DF,George AJ

更新日期：1999-05-01 00:00:00

abstract：:Polyethylenimine (PEI) has been studied as an efficient nonviral gene transfer vector. Here, we report the biodistribution fates and safety of plasmid DNA intravenously administered in PEI complexes. Using pCMVbeta as a model gene, the biodistribution of plasmid DNA was measured by quantitative polymerase chain reacti...

journal_title：Gene therapy

authors： Oh YK,Kim JP,Yoon H,Kim JM,Yang JS,Kim CK

更新日期：2001-10-01 00:00:00

abstract：:Urea cycle defects presenting in the neonatal period with hyperammonaemia are associated with high morbidity and mortality, and necessitate liver transplantation for long-term management. Gene therapy is therefore an attractive possibility, with vectors based on adeno-associated virus (rAAV) currently showing exciting...

journal_title：Gene therapy

authors： Cunningham SC,Kok CY,Spinoulas A,Carpenter KH,Alexander IE

更新日期：2013-12-01 00:00:00

abstract：:Current tools for the inhibition of microRNA (miR) function are limited to modified antisense oligonucleotides, sponges and decoy RNA molecules and none have been used to understand miR function during development. CRISPR/Cas-mediated deletion of miR sequences within the genome requires multiple chromosomal deletions ...

journal_title：Gene therapy

authors： Cao H,Yu W,Li X,Wang J,Gao S,Holton NE,Eliason S,Sharp T,Amendt BA

更新日期：2016-06-01 00:00:00

abstract：:Bone marrow presents an attractive option for the treatment of articular cartilage defects as it is readily accessible, it contains mesenchymal progenitor cells that can undergo chondrogenic differentiation and, once coagulated, it provides a natural scaffold that contains the cells within the defect. This study was p...

journal_title：Gene therapy

authors： Ivkovic A,Pascher A,Hudetz D,Maticic D,Jelic M,Dickinson S,Loparic M,Haspl M,Windhager R,Pecina M

更新日期：2010-06-01 00:00:00

abstract：:Development of efficient short-term gene transfer technologies for embryonic stem (ES) cells is urgently needed for various existing and new ES cell-based research strategies. In this study, we present a highly efficient, nonviral non-DNA technology for genetic loading of mouse ES cells based on electroporation of def...

journal_title：Gene therapy

authors： Ponsaerts P,Brown JP,Van den Plas D,Van den Eeden L,Van Bockstaele DR,Jorens PG,Van Tendeloo VF,Merregaert J,Singh PB,Berneman ZN

更新日期：2004-11-01 00:00:00

abstract：:We constructed a melanoma-specific oncolytic adenoviral vector Ad.MCDIRESE1.71Hsp3, in which the cytosine deaminase and adenoviral E1A genes linked by the IRES sequence were under the control of a mouse tyrosinase enhancer/promoter transcriptional element in the E1 region of the vector. We also inserted the human heat...

journal_title：Gene therapy

authors： Liu Y,Ye T,Sun D,Maynard J,Deisseroth A

更新日期：2006-08-01 00:00:00

abstract：:Lentivirus can be engineered to be a highly potent vector for gene therapy applications. However, generation of clinical grade vectors in enough quantities for therapeutic use is still troublesome and limits the preclinical and clinical experiments. As a first step to solve this unmet need we recently introduced a bac...

journal_title：Gene therapy

authors： Lesch HP,Laitinen A,Peixoto C,Vicente T,Makkonen KE,Laitinen L,Pikkarainen JT,Samaranayake H,Alves PM,Carrondo MJ,Ylä-Herttuala S,Airenne KJ

更新日期：2011-06-01 00:00:00

abstract：:Human adipose tissue mesenchymal stromal cells (AMSCs) share common traits, including similar differentiation potential and cell surface markers, with their bone marrow counterparts. Owing to their general availability, higher abundance and ease of isolation AMSCs may be convenient autologous delivery vehicles for loc...

journal_title：Gene therapy

authors： Vilalta M,Dégano IR,Bagó J,Aguilar E,Gambhir SS,Rubio N,Blanco J

更新日期：2009-04-01 00:00:00

abstract：:Coagulation factor X (FX)-binding ablated adenovirus type 5 (Ad5) vectors have been genetically engineered to ablate the interaction with FX, resulting in substantially reduced hepatocyte transduction following intravenous administration in rodents. Here, we quantify viral genomes and gene transfer mediated by Ad5 and...

journal_title：Gene therapy

authors： Alba R,Bradshaw AC,Mestre-Francés N,Verdier JM,Henaff D,Baker AH

更新日期：2012-01-01 00:00:00

abstract：:Bone morphogenetic protein (BMP) adenoviral vectors for the induction of osteogenesis are being developed for the treatment of bone pathology. However, it is still unknown which BMP adenoviral vector has the highest potential to stimulate bone formation in vivo. In this study, the osteogenic activities of recombinant ...

journal_title：Gene therapy

authors： Li JZ,Li H,Sasaki T,Holman D,Beres B,Dumont RJ,Pittman DD,Hankins GR,Helm GA

更新日期：2003-09-01 00:00:00