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Items: 1 to 20 of 357


Development of Methods for the Selective Measurement of the Single Amino Acid Exchange Variant Coagulation Factor IX Padua.

Weber A, Engelmaier A, Voelkel D, Pachlinger R, Scheiflinger F, Monahan PE, Rottensteiner H.

Mol Ther Methods Clin Dev. 2018 Jun 28;10:29-37. doi: 10.1016/j.omtm.2018.05.004. eCollection 2018 Sep 21.


Integrated Human Evaluation of the Lysophosphatidic Acid Pathway as a Novel Therapeutic Target in Atherosclerosis.

Aldi S, Matic LP, Hamm G, van Keulen D, Tempel D, Holmstrøm K, Szwajda A, Nielsen BS, Emilsson V, Ait-Belkacem R, Lengquist M, Paulsson-Berne G, Eriksson P, Lindeman JHN, Gool AJ, Stauber J, Hedin U, Hurt-Camejo E.

Mol Ther Methods Clin Dev. 2018 Jun 27;10:17-28. doi: 10.1016/j.omtm.2018.05.003. eCollection 2018 Sep 21.


GMP Production and Scale-Up of Adherent Neural Stem Cells with a Quantum Cell Expansion System.

Tirughana R, Metz MZ, Li Z, Hall C, Hsu D, Beltzer J, Annala AJ, Oganesyan D, Gutova M, Aboody KS.

Mol Ther Methods Clin Dev. 2018 Jul 7;10:48-56. doi: 10.1016/j.omtm.2018.05.006. eCollection 2018 Sep 21.


A Recombinant Baculovirus Efficiently Generates Recombinant Adeno-Associated Virus Vectors in Cultured Insect Cells and Larvae.

Wu Y, Jiang L, Geng H, Yang T, Han Z, He X, Lin K, Xu F.

Mol Ther Methods Clin Dev. 2018 Jul 4;10:38-47. doi: 10.1016/j.omtm.2018.05.005. eCollection 2018 Sep 21.


Targeting Heparan Sulfate Proteoglycans as a Novel Therapeutic Strategy for Mucopolysaccharidoses.

De Pasquale V, Sarogni P, Pistorio V, Cerulo G, Paladino S, Pavone LM.

Mol Ther Methods Clin Dev. 2018 Jun 18;10:8-16. doi: 10.1016/j.omtm.2018.05.002. eCollection 2018 Sep 21.


Serum-free Erythroid Differentiation for Efficient Genetic Modification and High-Level Adult Hemoglobin Production.

Uchida N, Demirci S, Haro-Mora JJ, Fujita A, Raines LN, Hsieh MM, Tisdale JF.

Mol Ther Methods Clin Dev. 2018 Mar 22;9:247-256. doi: 10.1016/j.omtm.2018.03.007. eCollection 2018 Jun 15.


A Rationally Engineered Capsid Variant of AAV9 for Systemic CNS-Directed and Peripheral Tissue-Detargeted Gene Delivery in Neonates.

Wang D, Li S, Gessler DJ, Xie J, Zhong L, Li J, Tran K, Van Vliet K, Ren L, Su Q, He R, Goetzmann JE, Flotte TR, Agbandje-McKenna M, Gao G.

Mol Ther Methods Clin Dev. 2018 Mar 16;9:234-246. doi: 10.1016/j.omtm.2018.03.004. eCollection 2018 Jun 15.


Cre Recombinase Mediates the Removal of Bacterial Backbone to Efficiently Generate rSV40.

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

Mol Ther Methods Clin Dev. 2018 Feb 27;9:225-233. doi: 10.1016/j.omtm.2018.02.010. eCollection 2018 Jun 15.


Seizure-Suppressant and Neuroprotective Effects of Encapsulated BDNF-Producing Cells in a Rat Model of Temporal Lobe Epilepsy.

Falcicchia C, Paolone G, Emerich DF, Lovisari F, Bell WJ, Fradet T, Wahlberg LU, Simonato M.

Mol Ther Methods Clin Dev. 2018 Mar 9;9:211-224. doi: 10.1016/j.omtm.2018.03.001. eCollection 2018 Jun 15.


A Non-integrating Lentiviral Approach Overcomes Cas9-Induced Immune Rejection to Establish an Immunocompetent Metastatic Renal Cancer Model.

Hu J, Schokrpur S, Archang M, Hermann K, Sharrow AC, Khanna P, Novak J, Signoretti S, Bhatt RS, Knudsen BS, Xu H, Wu L.

Mol Ther Methods Clin Dev. 2018 Feb 23;9:203-210. doi: 10.1016/j.omtm.2018.02.009. eCollection 2018 Jun 15.


Efficacy and Safety of Glycosidic Enzymes for Improved Gene Delivery to the Retina following Intravitreal Injection in Mice.

Cehajic-Kapetanovic J, Milosavljevic N, Bedford RA, Lucas RJ, Bishop PN.

Mol Ther Methods Clin Dev. 2017 Dec 22;9:192-202. doi: 10.1016/j.omtm.2017.12.002. eCollection 2018 Jun 15.


Development of Intrathecal AAV9 Gene Therapy for Giant Axonal Neuropathy.

Bailey RM, Armao D, Nagabhushan Kalburgi S, Gray SJ.

Mol Ther Methods Clin Dev. 2018 Feb 15;9:160-171. doi: 10.1016/j.omtm.2018.02.005. eCollection 2018 Jun 15.


Isoliquiritigenin Inhibits IL-1β-Induced Production of Matrix Metalloproteinase in Articular Chondrocytes.

Zhang L, Ma S, Su H, Cheng J.

Mol Ther Methods Clin Dev. 2018 Feb 15;9:153-159. doi: 10.1016/j.omtm.2018.02.006. eCollection 2018 Jun 15.


Integrating HDAd5/35++ Vectors as a New Platform for HSC Gene Therapy of Hemoglobinopathies.

Li C, Psatha N, Wang H, Singh M, Samal HB, Zhang W, Ehrhardt A, Izsvák Z, Papayannopoulou T, Lieber A.

Mol Ther Methods Clin Dev. 2018 Feb 15;9:142-152. doi: 10.1016/j.omtm.2018.02.004. eCollection 2018 Jun 15.


Adeno-associated Virus Genome Population Sequencing Achieves Full Vector Genome Resolution and Reveals Human-Vector Chimeras.

Tai PWL, Xie J, Fong K, Seetin M, Heiner C, Su Q, Weiand M, Wilmot D, Zapp ML, Gao G.

Mol Ther Methods Clin Dev. 2018 Feb 13;9:130-141. doi: 10.1016/j.omtm.2018.02.002. eCollection 2018 Jun 15.


Influence of Pre-existing Anti-capsid Neutralizing and Binding Antibodies on AAV Vector Transduction.

Fitzpatrick Z, Leborgne C, Barbon E, Masat E, Ronzitti G, van Wittenberghe L, Vignaud A, Collaud F, Charles S, Simon Sola M, Jouen F, Boyer O, Mingozzi F.

Mol Ther Methods Clin Dev. 2018 Feb 13;9:119-129. doi: 10.1016/j.omtm.2018.02.003. eCollection 2018 Jun 15.


Transduction Patterns of Adeno-associated Viral Vectors in a Laser-Induced Choroidal Neovascularization Mouse Model.

Lee SH, Kim YS, Nah SK, Kim HJ, Park HY, Yang JY, Park K, Park TK.

Mol Ther Methods Clin Dev. 2018 Jan 31;9:90-98. doi: 10.1016/j.omtm.2018.01.008. eCollection 2018 Jun 15.


Amelioration of Muscle and Nerve Pathology in LAMA2 Muscular Dystrophy by AAV9-Mini-Agrin.

Qiao C, Dai Y, Nikolova VD, Jin Q, Li J, Xiao B, Li J, Moy SS, Xiao X.

Mol Ther Methods Clin Dev. 2018 Jan 31;9:47-56. doi: 10.1016/j.omtm.2018.01.005. eCollection 2018 Jun 15.


The Biological Activity of AAV Vectors for Choroideremia Gene Therapy Can Be Measured by In Vitro Prenylation of RAB6A.

Patrício MI, Barnard AR, Cox CI, Blue C, MacLaren RE.

Mol Ther Methods Clin Dev. 2018 Mar 28;9:288-295. doi: 10.1016/j.omtm.2018.03.009. eCollection 2018 Jun 15.


Enhanced Production of Exosome-Associated AAV by Overexpression of the Tetraspanin CD9.

Schiller LT, Lemus-Diaz N, Rinaldi Ferreira R, Böker KO, Gruber J.

Mol Ther Methods Clin Dev. 2018 Mar 29;9:278-287. doi: 10.1016/j.omtm.2018.03.008. eCollection 2018 Jun 15.

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