Therapeutic efficacy of PUMA for malignant glioma cells regardless of p53 status.

abstract：:Adeno-associated virus (AAV) vector technology is rapidly advancing and becoming not only the leading vector platform in the field of gene therapy but also a useful tool for functional genomic studies of novel proteins. As most vectors utilize constitutive promoters, this results in transgene expression during product...

journal_title：Human gene therapy

authors： Guimaro MC,Afione SA,Tanaka T,Chiorini JA

更新日期：2020-10-01 00:00:00

abstract：:The first report of in vivo gene delivery to the retina dates back to 1987 when a retroviral vector was injected intraocularly in newborn mice. Later came the observation that retinal cells could be successfully transduced using adenoviral and then adeno-associated and lentiviral vectors. By 2000, it had become clear ...

journal_title：Human gene therapy

authors： Auricchio A,Smith AJ,Ali RR

更新日期：2017-11-01 00:00:00

abstract：:Leukocyte adhesion deficiency type I (LAD-I) is a primary immunodeficiency caused by mutations in the ITGB2 gene and is characterized by recurrent and life-threatening bacterial infections. These mutations lead to defective or absent expression of β2 integrins on the leukocyte surface, compromising adhesion and extrav...

journal_title：Human gene therapy

authors： Leon-Rico D,Aldea M,Sanchez-Baltasar R,Mesa-Nuñez C,Record J,Burns SO,Santilli G,Thrasher AJ,Bueren JA,Almarza E

更新日期：2016-09-01 00:00:00

abstract：:Isolated methylmalonic acidemia (MMA), a group of autosomal recessive inborn errors of metabolism, is most commonly caused by complete (mut(0)) or partial (mut(-)) deficiency of the enzyme methylmalonyl-CoA mutase (MUT). The severe metabolic instability and increased mortality experienced by many affected individuals,...

journal_title：Human gene therapy

authors： Harrington EA,Sloan JL,Manoli I,Chandler RJ,Schneider M,McGuire PJ,Calcedo R,Wilson JM,Venditti CP

更新日期：2016-05-01 00:00:00

abstract：:Immunologically sensitized recipients present one of the most critical problems in clinical organ transplantation today, since preformed antibodies rapidly destroy donor tissue expressing specific MHC class I antigens (Ag). Therefore, sensitized patients are either unable to receive a compatible organ, or experience a...

journal_title：Human gene therapy

authors： Geissler EK,Graeb C,Tange S,Guba M,Jauch KW,Scherer MN

更新日期：2000-02-10 00:00:00

abstract：:We reported total correction of blood coagulation plasma factor VIII (FVIII) activity, using adeno-associated virus serotype 8 (AAV8) vectors for liver-specific gene transfer in hemophilia A mice. We now show, irrespective of immunosuppression or route of administration, total long-term correction of hemophilia A mice...

journal_title：Human gene therapy

authors： Sarkar R,Mucci M,Addya S,Tetreault R,Bellinger DA,Nichols TC,Kazazian HH Jr

更新日期：2006-04-01 00:00:00

abstract：:Despite improvements in drug and device therapy for heart failure, hospitalization rates and mortality have changed little in the past decade. Randomized clinical trials using gene transfer to improve function of the failing heart are the focus of this review. Four randomized clinical trials of gene transfer in heart ...

journal_title：Human gene therapy

authors： Penny WF,Hammond HK

更新日期：2017-05-01 00:00:00

abstract：:Recombinant adeno-associated virus 2 (rAAV) vectors have been successfully used for sustained expression of therapeutic genes. The potential of using rAAV as a cancer vaccine vector and the impact of a bacterial plasmid adjuvant on this activity were investigated. C57BL/6 mice received a single intramuscular injection...

journal_title：Human gene therapy

authors： Ponnazhagan S,Mahendra G,Lima J,Aldrich WA,Jenkins CB,Ren C,Kumar S,Kallman L,Strong TV,Shaw DR,Triozzi PL

更新日期：2004-09-01 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：: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：:Naked plasmid DNA electrotransfer offers advantages over viral-based gene delivery, including being regulatory permissive, but factors influencing expression efficiency and cell fate impact on translational utility. This study compared co-expression of red and green fluorescence reporter plasmids with differing promot...

journal_title：Human gene therapy

authors： Pinyon JL,Klugmann M,Lovell NH,Housley GD

更新日期：2019-02-01 00:00:00

abstract：:Neurodegeneration in Parkinson's disease (PD) affects mainly dopaminergic neurons in the substantia nigra, where age-related, increasing percentages of cells lose detectable respiratory activity associated with depletion of intact mitochondrial DNA (mtDNA). Replenishment of mtDNA might improve neuronal bioenergetic fu...

journal_title：Human gene therapy

authors： Keeney PM,Quigley CK,Dunham LD,Papageorge CM,Iyer S,Thomas RR,Schwarz KM,Trimmer PA,Khan SM,Portell FR,Bergquist KE,Bennett JP Jr

更新日期：2009-08-01 00:00:00

abstract：:RNA interference (RNAi) is an evolutionarily conserved mechanism of posttranscriptional gene-specific silencing. For in vivo applications, RNAi has been hampered until recently by inefficient delivery methods and by the transient nature of the gene suppression. Lentiviral vectors (LVs) hold great promise for gene ther...

journal_title：Human gene therapy

authors： Van den Haute C,Eggermont K,Nuttin B,Debyser Z,Baekelandt V

更新日期：2003-12-10 00:00:00

abstract：:Recombinant adeno-associated virus (AAV)-based vectors expressing therapeutic gene products have shown great potential for human gene therapy. One major challenge for translation of promising research to clinical development is the manufacture of sufficient quantities of AAV vectors that meet stringent standards for p...

journal_title：Human gene therapy

authors： Wright JF

更新日期：2009-07-01 00:00:00

abstract：:Adenovirus vectors transduce liver hepatocytes with extreme efficiency; however, transgene expression is eliminated within 2 weeks. Extinction of transgene expression has been attributed to infiltrating cytotoxic T lymphocytes (CTLs) in the liver in a process that resembles a number of human diseases, including viral ...

journal_title：Human gene therapy

authors： Chirmule N,Moscioni AD,Qian Y,Qian R,Chen Y,Wilson JM

更新日期：1999-01-20 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：:Expression of a gene encoding the diphtheria toxin A (DT-A) fragment, controlled by tissue specific regulatory elements, has previously been used to kill selected cell populations. Here, we have examined the feasibility of controlling DT-A expression using regulatory systems from the human immunodeficiency virus (HIV-...

journal_title：Human gene therapy

authors： Harrison GS,Maxwell F,Long CJ,Rosen CA,Glode LM,Maxwell IH

更新日期：1991-04-01 00:00:00

abstract：:Protein transduction domains (PTD), which can transport proteins or peptides across biological membranes, have been identified in several proteins of viral, invertebrate, and vertebrate origin. Here, we evaluate the immunological and biological consequences of including PTD in synthetic peptides and in DNA vaccines th...

journal_title：Human gene therapy

authors： Leifert JA,Lindencrona JA,Charo J,Whitton JL

更新日期：2001-10-10 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：:The in vitro genetic manipulation of dendritic cells (DCs) for the expression of foreign proteins or peptides will assist in the development of immunotherapeutic approaches to treat cancer, immunological disorders, and/or infectious diseases. Reports have shown the expansion and differentiation of CD34(+) progenitor c...

journal_title：Human gene therapy

authors： Evans JT,Cravens P,Lipsky PE,Garcia JV

更新日期：2000-12-10 00:00:00

abstract：:Huntington's disease (HD) is a fatal neurodegenerative disease caused by a genetic expansion of the CAG repeat region in the huntingtin (HTT) gene. Studies in HD mouse models have shown that artificial miRNAs can reduce mutant HTT, but evidence for their effectiveness and safety in larger animals is lacking. HD transg...

journal_title：Human gene therapy

authors： Pfister EL,DiNardo N,Mondo E,Borel F,Conroy F,Fraser C,Gernoux G,Han X,Hu D,Johnson E,Kennington L,Liu P,Reid SJ,Sapp E,Vodicka P,Kuchel T,Morton AJ,Howland D,Moser R,Sena-Esteves M,Gao G,Mueller C,DiFiglia M

更新日期：2018-06-01 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：:Introduction of a new vaccine requires choosing a delivery system that provides safe administration and the desired level of immunogenicity. The safety, tolerability, and immunogenicity of three monthly 2.5-mg doses of a PfCSP DNA vaccine were evaluated in healthy volunteers as administered intramuscularly (IM) by nee...

journal_title：Human gene therapy

authors： Epstein JE,Gorak EJ,Charoenvit Y,Wang R,Freydberg N,Osinowo O,Richie TL,Stoltz EL,Trespalacios F,Nerges J,Ng J,Fallarme-Majam V,Abot E,Goh L,Parker S,Kumar S,Hedstrom RC,Norman J,Stout R,Hoffman SL

更新日期：2002-09-01 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：:Systemic administration of currently manufactured viral stocks has not so far achieved sufficient circulating titers to allow therapeutic targeting of metastatic disease. This is due to low initial viral titers, immune inactivation, nonspecific adhesion, and loss of particles. One way to exploit the elegant molecular ...

journal_title：Human gene therapy

authors： Harrington K,Alvarez-Vallina L,Crittenden M,Gough M,Chong H,Diaz RM,Vassaux G,Lemoine N,Vile R

更新日期：2002-07-20 00:00:00

abstract：:Immunotherapy with whole cell cancer vaccines has been tested in various tumor types. This study investigated the safety profile and antitumor activity of an allogeneic prostate carcinoma cell line, LNCaP, expressing recombinant human interleukin-2 and human interferon-gamma. Thirty HLA-A*0201-matched patients with pr...

journal_title：Human gene therapy

authors： Brill TH,Kübler HR,Pohla H,Buchner A,Fend F,Schuster T,van Randenborgh H,Paul R,Kummer T,Plank C,Eisele B,Breul J,Hartung R,Schendel DJ,Gansbacher B

更新日期：2009-12-01 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：: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：:The immunogenicity of adenovirus vectors remains a major obstacle to their safe and efficacious use for gene therapy. In order to identify T-cell epitopes directly from adenoviruses, four viral protein sequences were screened for the well-characterized 9-mer HLA-A2 binding motif. Peripheral blood mononuclear cells (PB...

journal_title：Human gene therapy

authors： Olive M,Eisenlohr L,Flomenberg N,Hsu S,Flomenberg P

更新日期：2002-07-01 00:00:00

abstract：:Extension of in vivo nucleic acid transfection techniques and increased information about those transfection properties and side effects are urgently needed to advance biological research and drug therapy. Tissue pressure-mediated transfection, involving lightly pressing the target tissue after intravenous injection o...

journal_title：Human gene therapy

authors： Mukai H,Kawakami S,Kamiya Y,Ma F,Takahashi H,Satake K,Terao K,Kotera H,Yamashita F,Hashida M

更新日期：2009-10-01 00:00:00