Human fetal liver cells for regulated ex vivo erythropoietin gene therapy.

abstract：:Human immunodeficiency virus (HIV) is an attractive target for chimeric antigen receptor (CAR) therapy. CAR T cells have proved remarkably potent in targeted killing of cancer cells, and we surmised that CAR T cells could prove useful in eradicating HIV-infected cells. Toward this goal, we interrogate several neutrali...

journal_title：Molecular therapy. Methods & clinical development

authors： Urak RZ,Soemardy C,Ray R,Li S,Shevchenko G,Scott T,Lim L,Wang X,Morris KV

更新日期：2020-09-28 00:00:00

abstract：:In vivo imaging of vector transgene expression would be particularly valuable for repetitive monitoring of therapy in the brain, where invasive tissue sampling is contraindicated. We evaluated adeno-associated virus vector expression of a dopamine-2 receptor (D2R) mutant (D2R80A) by positron emission tomography in the...

journal_title：Molecular therapy. Methods & clinical development

authors： Yoon SY,Gay-Antaki C,Ponde DE,Poptani H,Vite CH,Wolfe JH

更新日期：2014-06-04 00:00:00

abstract：:Hemophilia A, caused by a deficiency in factor VIII (FVIII), is the most severe inherited bleeding disorder. Hemophilia A is an attractive gene therapy candidate because even small increases in FVIII levels will positively alter the phenotype. While several vectors are under investigation, gene addition from an integr...

journal_title：Molecular therapy. Methods & clinical development

authors： Staber JM,Pollpeter MJ,Arensdorf A,Sinn PL,Rutkowski DT,McCray PB Jr

更新日期：2014-09-10 00:00:00

abstract：:Preclinical studies have demonstrated that a single injection of an adeno-associated virus (AAV) vector into the cerebrospinal fluid (CSF) can achieve widespread gene transfer throughout the central nervous system. Successfully translating this approach to humans requires identifying factors that influence AAV distrib...

journal_title：Molecular therapy. Methods & clinical development

authors： Hinderer C,Katz N,Dyer C,Goode T,Johansson J,Bell P,Richman L,Buza E,Wilson JM

更新日期：2020-04-18 00:00:00

abstract：:Duchenne muscular dystrophy (DMD) is a fatal disease of striated muscle deterioration. A unique therapeutic approach for DMD is the use of synthetic membrane stabilizers to protect the fragile dystrophic sarcolemma against contraction-induced mechanical stress. Block copolymer-based membrane stabilizer poloxamer 188 (...

journal_title：Molecular therapy. Methods & clinical development

authors： Houang EM,Haman KJ,Filareto A,Perlingeiro RC,Bates FS,Lowe DA,Metzger JM

更新日期：2015-11-11 00:00:00

abstract：:Rituximab is a mouse/human chimeric monoclonal antibody targeted toward CD20. It is efficient as first-line therapy of CD20-positive B-cell malignancies. However, a large fraction of treated patients relapse with rituximab-resistant disease. So far, only modest progress has been made in treatment options for rituximab...

journal_title：Molecular therapy. Methods & clinical development

authors： Richter M,Yumul R,Saydaminova K,Wang H,Gough M,Baldessari A,Cattaneo R,Lee F,Wang CH,Jang H,Astier A,Gopal A,Carter D,Lieber A

更新日期：2016-03-30 00:00:00

abstract：:Adeno-associated virus (AAV) vectors have emerged as a safe and efficient gene therapy platform. One complication is that a significant amount of empty particles have always been generated as impurities during AAV vector production. However, the effects of such particles on AAV vector performance remain unclear. Here ...

journal_title：Molecular therapy. Methods & clinical development

authors： Wang Q,Dong B,Pokiniewski KA,Firrman J,Wu Z,Chin MP,Chen X,Liu L,Xu R,Diao Y,Xiao W

更新日期：2016-12-24 00:00:00

abstract：:Vaccinia virus (VACV) was successfully used as a vaccine in the smallpox eradication campaign. Since then, it has been widely used in the development of vaccine and therapeutic vectors. However, methods of generating and purifying recombinant VACVs (rVACVs) are often time-consuming, cumbersome, and in some cases requi...

journal_title：Molecular therapy. Methods & clinical development

authors： Jasperse B,O'Connell CM,Wang Y,Verardi PH

更新日期：2020-03-30 00:00:00

abstract：:Adeno-associated viruses (AAVs) are the vector of choice for gene therapy in the eye, and self-complementary AAVs (scAAVs), which do not require second-strand DNA synthesis, can be transduced into cells of the trabecular meshwork (TM). The scAAV transduction patterns in the anterior segment of normotensive eyes have b...

journal_title：Molecular therapy. Methods & clinical development

authors： Lee SH,Sim KS,Kim CY,Park TK

更新日期：2019-07-10 00:00:00

abstract：:The His723Arg (H723R) mutation in SLC26A4, encoding pendrin, is the most prevalent mutation in East Asia, resulting in DFNB4, an autosomal recessive type of genetic hearing loss. Although the main pathological mechanism of H723R was identified as a protein-folding defect in pendrin, there is still no curative treatmen...

journal_title：Molecular therapy. Methods & clinical development

authors： Choi HJ,Lee HJ,Choi JY,Jeon IH,Noh B,Devkota S,Lee HW,Eo SK,Choi JY,Lee MG,Jung J

更新日期：2019-11-30 00:00:00

abstract：:Herpes simplex keratitis (HSK) is the most common cause of corneal blindness in developed nations, caused by primary or recurrent herpes simplex virus 1 (HSV-1) infection of the cornea. Latent infection of HSV-1, especially in the trigeminal ganglion (TG), causes recurrence of HSV-1 infection. As antiviral treatment i...

journal_title：Molecular therapy. Methods & clinical development

authors： Chen Y,Zhi S,Liang P,Zheng Q,Liu M,Zhao Q,Ren J,Cui J,Huang J,Liu Y,Songyang Z

更新日期：2020-05-22 00:00:00

abstract：:The overall goal of our research is to establish a preformed molecular guidance pathway to direct the growth of dopaminergic axons from embryonic ventral mesencephalon (VM), tissue placed within the substantia nigra (SN), into the striatum to reconstruct the nigrostriatal pathway in a hemi-Parkinson's disease rat mode...

journal_title：Molecular therapy. Methods & clinical development

authors： Ghosh B,Zhang C,Ziemba KS,Fletcher AM,Yurek DM,Smith GM

更新日期：2019-07-11 00:00:00

abstract：:The tuberculosis (TB) vaccine MTBVAC is the only live-attenuated Mycobacterium tuberculosis (Mtb)-based vaccine in clinical development, and it confers superior protection in different animal models compared to the current vaccine, BCG (Mycobacterium bovis bacillus Calmette-Guérin). With the aim of using MTBVAC as a v...

journal_title：Molecular therapy. Methods & clinical development

authors： Broset E,Saubi N,Guitart N,Aguilo N,Uranga S,Kilpeläinen A,Eto Y,Hanke T,Gonzalo-Asensio J,Martín C,Joseph-Munné J

更新日期：2019-02-07 00:00:00

abstract：:Recombinant adeno-associated viral (rAAV) vectors have been widely used in human gene therapy. One major impediment to its broad application is the inability to produce high-quality vectors in mass quantity. Here, an efficient and scalable suspension cell culture system for the production of rAAV vectors is described....

journal_title：Molecular therapy. Methods & clinical development

authors： Wang Q,Wu Z,Zhang J,Firrman J,Wei H,Zhuang Z,Liu L,Miao L,Hu Y,Li D,Diao Y,Xiao W

更新日期：2017-11-07 00:00:00

abstract：:Recombinant adeno-associated virus (rAAV) vectors are considered ideal vehicles for human gene therapy. Meanwhile, non-viral strategies, such as transfection agents (TAs), have also shown promise to deliver genetic materials, such as siRNA. Transduction with the rAAV vector is performed concurrently with transfection ...

journal_title：Molecular therapy. Methods & clinical development

authors： Guo P,Yu C,Wang Q,Zhang R,Meng X,Feng Y

更新日期：2018-04-12 00:00:00

abstract：:We present an overview of clinical trials involving gene editing using clustered interspaced short palindromic repeats (CRISPR)-CRISPR-associated protein 9 (Cas9), transcription activator-like effector nucleases (TALENs), or zinc finger nucleases (ZFNs) and discuss the underlying mechanisms. In cancer immunotherapy, g...

journal_title：Molecular therapy. Methods & clinical development

authors： Ernst MPT,Broeders M,Herrero-Hernandez P,Oussoren E,van der Ploeg AT,Pijnappel WWMP

更新日期：2020-07-03 00:00:00

abstract：:To date, gene therapy with transiently derived lentivectors has been very successful to cure rare infant genetic diseases. However, transient manufacturing is unfeasible to treat adult malignancies because large vector lots are required. By contrast, stable manufacturing is the best option for high-incidence diseases ...

journal_title：Molecular therapy. Methods & clinical development

authors： Marin V,Stornaiuolo A,Piovan C,Corna S,Bossi S,Pema M,Giuliani E,Scavullo C,Zucchelli E,Bordignon C,Rizzardi GP,Bovolenta C

更新日期：2016-05-11 00:00:00

abstract：:Validation of gene transfer vectors containing tissue-specific promoters in cell-based functional assays poses a formidable challenge for gene therapy product development. Here, we describe a novel approach based on CRISPR/dCas9 transcriptional activation to achieve robust transgene expression from transgene cassettes...

journal_title：Molecular therapy. Methods & clinical development

authors： McDougald DS,Duong TT,Palozola KC,Marsh A,Papp TE,Mills JA,Zhou S,Bennett J

更新日期：2019-03-28 00:00:00

abstract：:Gene therapy has been shown to be a feasible approach to treat inherited disorders in vivo. Among the currently used viral vector systems, adeno-associated virus (AAV) vectors are the most advanced and have been applied in patients successfully. An important drawback of non-integrating AAV vectors is their loss of exp...

journal_title：Molecular therapy. Methods & clinical development

authors： Shi X,Ykema MR,Hazenoot J,Ten Bloemendaal L,Mancini I,Odijk M,de Haan P,Bosma PJ

更新日期：2018-02-27 00:00:00

abstract：:Recombinant adeno-associated virus (rAAV) is currently the best vector for gene delivery into the skeletal muscle. However, the 5-kb packaging size of this virus is a major obstacle for large gene transfer. This past decade, many different strategies were developed to circumvent this issue (concatemerization-splicing,...

journal_title：Molecular therapy. Methods & clinical development

authors： Pryadkina M,Lostal W,Bourg N,Charton K,Roudaut C,Hirsch ML,Richard I

更新日期：2015-03-25 00:00:00

abstract：:Extracellular vesicles (EVs) are membranous structures that protect RNAs from damage when circulating in complex biological fluids, such as plasma. RNAs are extremely specific to health and disease, being powerful tools for diagnosis, treatment response monitoring, and development of new therapeutic strategies for sev...

journal_title：Molecular therapy. Methods & clinical development

authors： Gaspar LS,Santana MM,Henriques C,Pinto MM,Ribeiro-Rodrigues TM,Girão H,Nobre RJ,Pereira de Almeida L

更新日期：2020-07-15 00:00:00

abstract：:Pompe disease (PD) is a lysosomal disorder caused by acid α-glucosidase (GAA) deficiency. Progressive muscular weakness is the major symptom of PD, and enzyme replacement therapy can improve the clinical outcome. However, to achieve a better clinical outcome, alternative therapeutic strategies are being investigated, ...

journal_title：Molecular therapy. Methods & clinical development

authors： Sato Y,Kobayashi H,Higuchi T,Shimada Y,Ida H,Ohashi T

更新日期：2016-08-10 00:00:00

abstract：:Recombinant adeno-associated virus (rAAV)rh74.MCK.GALGT2 is a muscle-specific gene therapy that is being developed to treat forms of muscular dystrophy. Here we report on an isolated limb infusion technique in a non-human primate model, where hindlimb blood flow is transiently isolated using balloon catheters to conce...

journal_title：Molecular therapy. Methods & clinical development

authors： Xu R,Jia Y,Zygmunt DA,Cramer ML,Crowe KE,Shao G,Maki AE,Guggenheim HN,Hood BC,Griffin DA,Peterson E,Bolon B,Cheatham JP,Cheatham SL,Flanigan KM,Rodino-Klapac LR,Chicoine LG,Martin PT

更新日期：2018-07-14 00:00:00

abstract：:Insertional mutagenesis has been associated with malignant cell transformation in gene therapy protocols, leading to discussions about vector security. Therefore, clonal analysis is important for the assessment of vector safety and its impact on patient health. Here, we report a unique approach to assess dynamic chang...

journal_title：Molecular therapy. Methods & clinical development

authors： Zanatta DB,Tsujita M,Borelli P,Aguiar RB,Ferrari DG,Strauss BE

更新日期：2014-11-19 00:00:00

abstract：:There is no effective serologic parameter to distinguish different types of pancreatitis now. To distinguish between acute pancreatitis (AP) and acute exacerbations of chronic pancreatitis (CP) and to determine whether fibrosis occurs in CP, we evaluated the ability to produce white blood cells (WBCs), the neutrophil-...

journal_title：Molecular therapy. Methods & clinical development

authors： Luo L,Zhang J,Yang J,Zhang H,Tang Y,Yang D,Dong H,Wu Y,Wang H,Ni B,Tian Z

更新日期：2020-05-22 00:00:00

abstract：:Endotoxin is the most common contaminant found in protein samples. Even a small amount of endotoxin can induce strong allergic reaction and death of a host organism. Endotoxin is also often detected in recombinant adeno-associated virus (rAAV) stocks prepared in research laboratories using off-the-shelf reagents; puri...

journal_title：Molecular therapy. Methods & clinical development

authors： Kondratova L,Kondratov O,Ragheb R,Zolotukhin S

更新日期：2019-09-06 00:00:00

abstract：:Lentiviral vectors (LVs) are increasingly employed in gene and cell therapy. Standard laboratory production of LVs is not easily scalable, and research-grade LVs often contain contaminants that can interfere with downstream applications. Moreover, purified LV production pipelines have been developed mainly for costly,...

journal_title：Molecular therapy. Methods & clinical development

authors： Soldi M,Sergi Sergi L,Unali G,Kerzel T,Cuccovillo I,Capasso P,Annoni A,Biffi M,Rancoita PMV,Cantore A,Lombardo A,Naldini L,Squadrito ML,Kajaste-Rudnitski A

更新日期：2020-10-20 00:00:00

abstract：:Identification and characterization of disease-associated variants in monogenic disorders is an important aspect of diagnosis, genetic counseling, prediction of disease severity, and development of therapy. However, the effects of disease-associated variants on pre-mRNA splicing and mRNA degradation are difficult to p...

journal_title：Molecular therapy. Methods & clinical development

authors： Broeders M,Smits K,Goynuk B,Oussoren E,van den Hout HJMP,Bergsma AJ,van der Ploeg AT,Pijnappel WWMP

更新日期：2020-09-16 00:00:00

abstract：:Lentiviral vectors have emerged as an efficient, safe therapeutic tool for gene therapy based on hematopoietic stem cells (HSCs) or T cells. However, the monitoring of transduced cells in preclinical models remains challenging because of the inefficient transduction of murine primary T cells with lentiviral vectors, i...

journal_title：Molecular therapy. Methods & clinical development

authors： Delville M,Soheili T,Bellier F,Durand A,Denis A,Lagresle-Peyrou C,Cavazzana M,Andre-Schmutz I,Six E

更新日期：2018-08-08 00:00:00

abstract：:Because many peptide and peptide-mimetic drugs are substrates of peptide transporter 1, it is important to evaluate the peptide transporter 1-mediated intestinal absorption of drug candidates in the early phase of drug development. Although intestinal cell lines treated with inhibitors of peptide transporter 1 are wid...

journal_title：Molecular therapy. Methods & clinical development

authors： Kawai K,Negoro R,Ichikawa M,Yamashita T,Deguchi S,Harada K,Hirata K,Takayama K,Mizuguchi H

更新日期：2019-11-21 00:00:00