Bone Marrow Mesenchymal Stem Cells-Derived Exosomal MicroRNA-150-3p Promotes Osteoblast Proliferation and Differentiation in Osteoporosis.

abstract：:Niemann-Pick type C (NP-C) disease is a neurodegenerative disorder characterized neuropathologically by ballooned neurons distended with lipid storage and widespread neuronal loss. Neural stem cells (NSC) derived from NP-C disease models have decreased ability for self-renewal and neuronal differentiation. Investigati...

journal_title：Human gene therapy

authors： Lee H,Kang JE,Lee JK,Bae JS,Jin HK

更新日期：2013-07-01 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：:Direct arterial gene transfer has been previously achieved using double-balloon catheters and perforated balloons, in most cases facilitated by the use of cationic liposomes or viral vectors. These gene delivery systems, however, have been compromised by issues relating to efficacy and/or safety, and furthermore requi...

journal_title：Human gene therapy

authors： Riessen R,Rahimizadeh H,Blessing E,Takeshita S,Barry JJ,Isner JM

更新日期：1993-12-01 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：: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：: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：:Somatic gene therapy for pulmonary diseases must be accomplished in vivo, requiring the spread of a gene transfer vector across a vast expanse of respiratory epithelium. Surfactant, a naturally occurring protein and lipid mixture used to treat the respiratory distress syndrome of prematurity, disperses rapidly and eve...

journal_title：Human gene therapy

authors： Katkin JP,Husser RC,Langston C,Welty SE

更新日期：1997-01-20 00:00:00

abstract：:We report a novel method for targeting adenovirus-mediated gene delivery. By irradiating mammalian cells prior to adenoviral transduction, adenoviral gene transfer is greatly improved and the adenoviral genome integrates into cellular DNA. In this work, human and rodent cell lines were irradiated and subsequently tran...

journal_title：Human gene therapy

authors： Zeng M,Cerniglia GJ,Eck SL,Stevens CW

更新日期：1997-06-10 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：: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：:Mucopolysaccharidosis type IIIA (MPSIIIA) is a rare lysosomal storage disorder caused by mutations in the sulfamidase gene. Accumulation of glycosaminoglycan (GAG) inside the lysosomes is associated with severe neurodegeneration as well as peripheral organ pathological changes leading to death of affected individuals ...

journal_title：Human gene therapy

authors： Ruzo A,Marcó S,García M,Villacampa P,Ribera A,Ayuso E,Maggioni L,Mingozzi F,Haurigot V,Bosch F

更新日期：2012-12-01 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：:Spinal muscular atrophy (SMA) is an autosomal recessive disease affecting ∼1 in 10,000 live births. The most striking component is the loss of α-motor neurons in the ventral horn of the spinal cord, resulting in progressive paralysis and eventually premature death. There is no current treatment paradigm other than sup...

journal_title：Human gene therapy

authors： Porensky PN,Burghes AH

更新日期：2013-05-01 00:00:00

abstract：:When transferring the human multidrug resistance 1 (MDR1) cDNA, FMEV retroviral vectors mediate high-dose multidrug resistance and, thus, background-free selection in primary human hematopoietic progenitor cells. Here, we analyzed strategies for co-expression of a second gene from an FMEV:MDR1 vector. When linking the...

journal_title：Human gene therapy

authors： Hildinger M,Fehse B,Hegewisch-Becker S,John J,Rafferty JR,Ostertag W,Baum C

更新日期：1998-01-01 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：:Targeted delivery of intravenously administered genetically altered cells or stem cells is still in an early stage of investigation. We developed a method of delivering iron oxide (ferumoxide)-labeled mesenchymal stem cells (MSCs) to a targeted area in an animal model by applying an external magnet. Rats with or witho...

journal_title：Human gene therapy

authors： Arbab AS,Jordan EK,Wilson LB,Yocum GT,Lewis BK,Frank JA

更新日期：2004-04-01 00:00:00

abstract：:Umbilical cord blood (CB) from the early gestational human fetus is recognized as a rich source of hematopoietic stem cells. To examine the value of fetal CB for gene therapy of inborn immunohematopoietic disorders, we tested the feasibility of genetic modification of CD34(+) cells from CB at weeks 24 to 34 of pregnan...

journal_title：Human gene therapy

authors： Luther-Wyrsch A,Costello E,Thali M,Buetti E,Nissen C,Surbek D,Holzgreve W,Gratwohl A,Tichelli A,Wodnar-Filipowicz A

更新日期：2001-03-01 00:00:00

abstract：:The initial stages following the in vitro cytokine stimulation of human cord blood CD34+ cells overlap with the period when lentiviral gene transfer is typically performed. Single-cell transcriptional profiling and time-lapse microscopy were used to investigate how the vector-cell crosstalk impacts on the fate decisio...

journal_title：Human gene therapy

authors： Moussy A,Papili Gao N,Corre G,Poletti V,Majdoul S,Fenard D,Gunawan R,Stockholm D,Páldi A

更新日期：2019-08-01 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：: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：:Production of clinical-grade gammaretroviral vectors for ex vivo gene delivery requires a scalable process that can rapidly generate large amounts of vector supernatant, clear large numbers of residual packaging cells with minimal decreases in vector titer, and satisfy all current regulatory guidelines regarding produ...

journal_title：Human gene therapy

authors： Feldman SA,Goff SL,Xu H,Black MA,Kochenderfer JN,Johnson LA,Yang JC,Wang Q,Parkhurst MR,Cross S,Morgan RA,Cornetta K,Rosenberg SA

更新日期：2011-01-01 00:00:00

abstract：:The use of gene-engineered T cells expressing chimeric single-chain (scFv) receptors capable of codelivering CD28 costimulation and T cell receptor zeta chain (TCR-zeta) activation signals has emerged as a promising treatment regimen for cancer. Using retroviral transduction, primary human T lymphocytes were gene-engi...

journal_title：Human gene therapy

authors： Teng MW,Kershaw MH,Moeller M,Smyth MJ,Darcy PK

更新日期：2004-07-01 00:00:00

abstract：:In a model of growth-restricted sheep pregnancy, it was previously demonstrated that transient uterine artery VEGF overexpression can improve fetal growth. This approach was tested in guinea-pig pregnancies, where placental physiology is more similar to humans. Fetal growth restriction (FGR) was attained through peri-...

journal_title：Human gene therapy

authors： Swanson AM,Rossi CA,Ofir K,Mehta V,Boyd M,Barker H,Ledwozyw A,Vaughan O,Martin J,Zachary I,Sebire N,Peebles DM,David AL

更新日期：2016-12-01 00:00:00

abstract：:Overexpression of human manganese-containing superoxide dismutase (MnSOD) activity has been demonstrated to suppress malignancy in human melanoma and breast carcinoma cells in vitro and in vivo. To study its effects on human oral squamous carcinoma cells, stable transfection and expression of MnSOD in SCC-25 cells hav...

journal_title：Human gene therapy

authors： Liu R,Oberley TD,Oberley LW

更新日期：1997-03-20 00:00:00

abstract：:Combining chemotherapy and immunotherapy is problematic because chemotherapy can ablate the immune responses initiated by modulators of the immune system. We hypothesized that protection of immunocompetent cells from the toxic effects of chemotherapy, using drug resistance gene therapy strategies, would allow the comb...

journal_title：Human gene therapy

authors： McMillin DW,Hewes B,Gangadharan B,Archer DR,Mittler RS,Spencer HT

更新日期：2006-08-01 00:00:00

abstract：:Serum-induced inactivation of retroviruses is the most critical limitation for in vivo gene transfer therapy. To solve this problem, we searched for reagents that protect retroviruses from inactivation. The effects of the protease inhibitors FOY-007 and FOY-305 and of an inhibitor of the complement pathway FUT-175, al...

journal_title：Human gene therapy

authors： Miyao Y,Ikenaka K,Kishima H,Tamura M,Nakamura K,Kurumi M,Hayakawa T,Shimizu K

更新日期：1997-09-01 00:00:00

abstract：:With the aim of developing new gene transfer tools for treating CF with gene therapy, we have synthesized a novel family of molecules named cationic phosphonolipids. The most efficient among them were selected by in vitro screening to compare their activities in vivo in mouse lungs. We used a reporter gene whose activ...

journal_title：Human gene therapy

authors： Guillaume-Gable C,Floch V,Mercier B,Audrézet MP,Gobin E,Le Bolch G,Yaouanc JJ,Clément JC,des Abbayes H,Leroy JP,Morin V,Férec C

更新日期：1998-11-01 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：: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：:To target expression of toxic genes to Epstein-Barr virus (EBV)-associated tumor cells, we have developed an EBV-driven enzyme prodrug system (EDEPS) that takes advantage of the trans-activating properties of EBNA1, a latent protein expressed in all EBV-containing cells, to direct expression of cytosine deaminase (CD)...

journal_title：Human gene therapy

authors： Judde JG,Spangler G,Magrath I,Bhatia K

更新日期：1996-03-20 00:00:00