Recombinant truncated dystrophin minigenes: construction, expression, and adenoviral delivery.

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

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：:Adeno-associated virus (AAV) gene transfer is a promising treatment for genetic abnormalities. Optimal AAV vectors are showing success in clinical trials. Gene transfer to skeletal muscle and liver is being explored as a potential therapy for some conditions, that is, α1-antitrypsin (AAT) disorder and hemophilia B. Ex...

journal_title：Human gene therapy

authors： Carrig S,Bijjiga E,Wopat MJ,Martino AT

更新日期：2016-11-01 00:00:00

abstract：:Clinical applications of gene therapy require advances in gene delivery systems. Although numerous clinical trials are already underway, the ultimate success of gene therapies will depend on gene transfer vectors that facilitate the expression of a specific gene at therapeutic levels in the desired cell populations wi...

journal_title：Human gene therapy

authors： Anderson DM,Hall LL,Ayyalapu AR,Irion VR,Nantz MH,Hecker JG

更新日期：2003-02-10 00:00:00

abstract：:The objective of this phase II investigation is to assess the safety and efficacy of a plasmid mediated approach to induce angiogenesis/arteriogenesis with the angiomatrix protein Del-1 (developmentally regulated endothelial locus 1), in subjects with intermittent claudication (IC) secondary to peripheral arterial dis...

journal_title：Human gene therapy

authors： Rajagopalan S,Olin JW,Young S,Erikson M,Grossman PM,Mendelsohn FO,Regensteiner JG,Hiatt WR,Annex BH

更新日期：2004-06-01 00:00:00

abstract：:Abstract Malignant gliomas (MGs) are highly vascularized, aggressive brain cancers carrying a dismal prognosis. Because of their high vascularity, anti-angiogenic therapy is a potential treatment option. Indeed, the anti-vascular endothelial growth factor (VEGF) antibody bevacizumab has demonstrated promising results ...

journal_title：Human gene therapy

authors： Samaranayake HD,Pikkarainen JT,Wirth T,Stedt H,Lesch HP,Dragneva G,Vuorio T,Määttä AM,Airenne K,Ylä-Herttuala S

更新日期：2014-11-01 00:00:00

abstract：:Unlike oncoretroviruses, lentiviral vectors can insert large genes and can target both dividing and nondividing cells; thus they hold unique promise as gene transfer agents. To enhance target range, the native lentiviral envelope glycoprotein is replaced (pseudotyped) with vesicular stomatitis virus G (VSVG), and the ...

journal_title：Human gene therapy

authors： Kumar M,Bradow BP,Zimmerberg J

更新日期：2003-01-01 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：:Human papillomavirus type 16 (HPV16) is associated with the development of anogenital cancers and their precursor lesions, intraepithelial neoplasia. Treatment strategies against HPV-induced intraepithelial neoplasia are not HPV specific and mostly consist of physical removal or ablation of lesions. We had previously ...

journal_title：Human gene therapy

authors： Sharma R,Palefsky JM

更新日期：2010-07-01 00:00:00

abstract：:Point mutations in the dystrophin gene cause dystrophin deficiency and muscular dystrophy in the mdx mouse and a subset of patients with Duchenne muscular dystrophy. As an approach to gene therapy for muscular dystrophies due to point mutations, we have studied the ability of RNA-DNA chimeric oligonucleotides (chimera...

journal_title：Human gene therapy

authors： Bertoni C,Rando TA

更新日期：2002-04-10 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：:Gene-modified replication-competent adenoviruses (Ads) are emerging as a promising new modality for the treatment of cancer. We have previously shown that E1B 19kDa and E1B 55kDa gene-deleted Ad (Ad-DeltaE1B19/55) exhibits improved tumor-specific replication and cell lysis, leading to an enhanced antitumor effect. In ...

journal_title：Human gene therapy

authors： Kim J,Kim JH,Choi KJ,Kim PH,Yun CO

更新日期：2007-09-01 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：: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：:The management of disorders of the nervous system remains a medical challenge. The key goals are to understand disease mechanisms, to validate therapeutic targets, and to develop new therapeutic strategies. Viral vector-mediated gene transfer can meet these goals and vectors based on lentiviruses have particularly use...

journal_title：Human gene therapy

authors： Wong LF,Goodhead L,Prat C,Mitrophanous KA,Kingsman SM,Mazarakis ND

更新日期：2006-01-01 00:00:00

abstract：:We previously reported that spliceosome-mediated RNA trans-splicing (SMaRT), using recombinant adenoviral vectors expressing pre-trans-splicing molecules (PTMs), could partially restore cystic fibrosis transmembrane conductance regulator (CFTR) chloride channel activity to polarized human DeltaF508 CF airway epithelia...

journal_title：Human gene therapy

authors： Liu X,Luo M,Zhang LN,Yan Z,Zak R,Ding W,Mansfield SG,Mitchell LG,Engelhardt JF

更新日期：2005-09-01 00:00:00

abstract：:Advanced prostate cancer is invariably lethal once it becomes androgen independent (AI). With the aim of developing a new treatment we have used the human androgen-independent prostate cancer cell line, PC-3, to evaluate the effectiveness of two enzyme-directed prodrug therapy (EPT) systems as a novel means for promot...

journal_title：Human gene therapy

authors： Martiniello-Wilks R,Garcia-Aragon J,Daja MM,Russell P,Both GW,Molloy PL,Lockett LJ,Russell PJ

更新日期：1998-07-20 00:00:00

abstract：:We report on an antitumor treatment involving electrogene therapy (EGT), a newly developed in vivo gene transfer method using electroporation. We carried out in vivo EGT in a subcutaneous model of CT26 colon carcinoma cells, using plasmid DNAs encoding interleukin 12 (IL-12) subunits. For this purpose, we developed tw...

journal_title：Human gene therapy

authors： Tamura T,Nishi T,Goto T,Takeshima H,Dev SB,Ushio Y,Sakata T

更新日期：2001-07-01 00:00:00

abstract：:Systemic administration of adenoviral vectors leads to activation of innate and antigen-specific immunity. In an attempt to diminish T and B cell-specific immune responses to E1-deleted adenoviral vectors, capsid proteins were modified with various activated monomethoxypolyethylene glycols (MPEGs). The impact of this ...

journal_title：Human gene therapy

authors： Croyle MA,Chirmule N,Zhang Y,Wilson JM

更新日期：2002-10-10 00:00:00

abstract：:We studied hematopoietic progenitors from fetal baboon blood, marrow, and liver at four time points (125, 140, 160, and 175 days) during the third trimester (gestation approximately 180 days) to determine if fetal baboons might be an appropriate model for in utero gene therapy of hematopoietic stem cells (HSCs). Cells...

journal_title：Human gene therapy

authors： Winkler A,Kiem HP,Shields LE,Sun QH,Andrews RG

更新日期：1999-03-01 00:00:00

abstract：:This multicenter phase I/II study evaluated the safety, pharmacokinetics, and antitumor effects of repeated doses of NV1020, a genetically engineered oncolytic herpes simplex virus, in patients with advanced metastatic colorectal cancer (mCRC). Patients with liver-dominant mCRC received four fixed NV1020 doses via wee...

journal_title：Human gene therapy

authors： Geevarghese SK,Geller DA,de Haan HA,Hörer M,Knoll AE,Mescheder A,Nemunaitis J,Reid TR,Sze DY,Tanabe KK,Tawfik H

更新日期：2010-09-01 00:00:00

abstract：:Aerosol delivery of adenoviral vectors is of particular interest in regard to gene therapy for cystic fibrosis (CF), with potential advantages of more uniform respiratory delivery, a less invasive approach, and ease of repetition. The AdHCMVsp1LacZ (AdLacZ) adenoviral vector was used to evaluate the feasibility of aer...

journal_title：Human gene therapy

authors： Katkin JP,Gilbert BE,Langston C,French K,Beaudet AL

更新日期：1995-08-01 00:00:00

abstract：:Mutations in the cilia-centrosomal protein CEP290 are frequently observed in autosomal recessive childhood blindness disorder Leber congenital amaurosis (LCA). No treatment or cure currently exists for this disorder. The Cep290rd16 (retinal degeneration 16) mouse (a model of LCA) carries a mutation in the Cep290 gene....

journal_title：Human gene therapy

authors： Zhang W,Li L,Su Q,Gao G,Khanna H

更新日期：2018-01-01 00:00:00

abstract：:Gene transfer of the herpes simplex virus thymidine kinase (TK) gene associated with ganciclovir (GCV) treatment can lead to death of TK-expressing cells, and of neighboring TK- cells because of the bystander effect. Thus, a small proportion of TK+ cells in a tumor can lead to its complete regression after GCV treatme...

journal_title：Human gene therapy

authors： Qiao J,Black ME,Caruso M

更新日期：2000-07-20 00:00:00

abstract：:Myelosuppression is the main side effect of cancer chemotherapy. An improved rate of retroviral vector-mediated gene transfer to hematopoietic stem cells, shown in more recent clinical trials, has created the basis to test the concept of myeloprotective gene therapy. We transplanted clinical-scale human peripheral blo...

journal_title：Human gene therapy

authors： Schiedlmeier B,Schilz AJ,Kühlcke K,Laufs S,Baum C,Zeller WJ,Eckert HG,Fruehauf S

更新日期：2002-01-20 00:00:00

abstract：:In summary, I will reiterate the five points I would like to leave with you today: First, the biological revolution has extraordinary power to do good. As long as the use of our new genetic knowledge is guided by the traditional ideals of the healing professions--to help improve the human condition without doing harm-...

journal_title：Human gene therapy

authors： Healy B

更新日期：1992-02-01 00:00:00

abstract：:Oncolytic measles virus (MV) encoding the human thyroidal sodium iodide symporter (MV-NIS) has proved to be safe after intraperitoneal or intravenous administration in patients with ovarian cancer or multiple myeloma, respectively, but it has not yet been administered through intratumoral injection in humans. Squamous...

journal_title：Human gene therapy

authors： Li H,Peng KW,Russell SJ

更新日期：2012-03-01 00:00:00

abstract：:Glioblastomas account for approximately 20% of all primary brain tumors in adults. Glioblastoma multiforme (GBM) is a highly malignant tumor. In spite of advances in surgery, chemotherapy, and radiotherapy, the life expectancy of the patient with glioblastoma is approximately 11 months. To enhance glioma-specific gene...

journal_title：Human gene therapy

authors： Ho IA,Lam PY,Hui KM

更新日期：2004-08-01 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：:For the successful application of RNA interference in vivo, it is desired to achieve (local) delivery of small interfering RNAs (siRNAs) and long-term gene silencing. Nonviral electrodelivery is suitable to obtain local and prolonged expression of transgenes. By intramuscular electrodelivery of a plasmid in which two ...

journal_title：Human gene therapy

authors： Eefting D,Grimbergen JM,de Vries MR,van Weel V,Kaijzel EL,Que I,Moon RT,Löwik CW,van Bockel JH,Quax PH

更新日期：2007-09-01 00:00:00