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Volume 19; Issue 12

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Molecular Therapy
Volume 19; Issue 12

Molecular Therapy

Volume 19; Issue 12

1

Potent and Selective Antisense Oligonucleotides Targeting Single-Nucleotide Polymorphisms in the Huntington Disease Gene / Allele-Specific Silencing of Mutant Huntingtin

Carroll, Jeffrey B, Warby, Simon C, Southwell, Amber L, Doty, Crystal N, Greenlee, Sarah, Skotte, Niels, Hung, Gene, Bennett, C Frank, Freier, Susan M, Hayden, Michael R

Journal:

Molecular Therapy

Année:

2011

Langue:

english

Fichier:

PDF, 311 KB

english, 2011

2

The Development of a Novel Cancer Immunotherapeutic Platform Using Tumor-targeting Mesenchymal Stem Cells and a Protein Vaccine

Wei, Hon-Jian, Wu, Alexander TH, Hsu, Chung-Huei, Lin, Yi-Ping, Cheng, Wen-Fang, Su, Ching-Hua, Chiu, Wen-Ta, Whang-Peng, Jacqueline, Douglas, Frank L, Deng, Win-Ping

Journal:

Molecular Therapy

Année:

2011

Langue:

english

Fichier:

PDF, 4.63 MB

english, 2011

3

Cytotoxic T Lymphocytes Simultaneously Targeting Multiple Tumor-associated Antigens to Treat EBV Negative Lymphoma

Gerdemann, Ulrike, Katari, Usha, Christin, Anne S, Cruz, Conrad R, Tripic, Tamara, Rousseau, Alexandra, Gottschalk, Stephen M, Savoldo, Barbara, Vera, Juan F, Heslop, Helen E, Brenner, Malcolm K, Boll

Journal:

Molecular Therapy

Année:

2011

Langue:

english

Fichier:

PDF, 484 KB

english, 2011

4

Influence of Cationic Lipid Composition on Gene Silencing Properties of Lipid Nanoparticle Formulations of siRNA in Antigen-Presenting Cells

Basha, Genc, Novobrantseva, Tatiana I, Rosin, Nicole, Tam, Yuen Yi C, Hafez, Ismail M, Wong, Matthew K, Sugo, Tsukasa, Ruda, Vera M, Qin, June, Klebanov, Boris, Ciufolini, Marco, Akinc, Akin, Tam, Yin

Journal:

Molecular Therapy

Année:

2011

Langue:

english

Fichier:

PDF, 4.82 MB

english, 2011

5

Uptake, Efficacy, and Systemic Distribution of Naked, Inhaled Short Interfering RNA (siRNA) and Locked Nucleic Acid (LNA) Antisense

Moschos, Sterghios A, Frick, Manfred, Taylor, Bruce, Turnpenny, Paul, Graves, Helen, Spink, Karen G, Brady, Kevin, Lamb, David, Collins, David, Rockel, Thomas D, Weber, Markus, Lazari, Ovadia, Perez-T

Journal:

Molecular Therapy

Année:

2011

Langue:

english

Fichier:

PDF, 1.36 MB

english, 2011

6

ATP7A Gene Addition to the Choroid Plexus Results in Long-term Rescue of the Lethal Copper Transport Defect in a Menkes Disease Mouse Model

Donsante, Anthony, Yi, Ling, Zerfas, Patricia M, Brinster, Lauren R, Sullivan, Patricia, Goldstein, David S, Prohaska, Joseph, Centeno, Jose A, Rushing, Elisabeth, Kaler, Stephen G

Journal:

Molecular Therapy

Année:

2011

Langue:

english

Fichier:

PDF, 4.38 MB

english, 2011

7

Preclinical Safety of RNAi-Mediated HTT Suppression in the Rhesus Macaque as a Potential Therapy for Huntington's Disease

McBride, Jodi L, Pitzer, Mark R, Boudreau, Ryan L, Dufour, Brett, Hobbs, Theodore, Ojeda, Sergio R, Davidson, Beverly L

Journal:

Molecular Therapy

Année:

2011

Langue:

english

Fichier:

PDF, 973 KB

english, 2011

8

lncRNAs: Finding the Forest Among the Trees?

Dinger, Marcel E

Journal:

Molecular Therapy

Année:

2011

Langue:

english

Fichier:

PDF, 269 KB

english, 2011

9

Systemic Administration of Combinatorial dsiRNAs via Nanoparticles Efficiently Suppresses HIV-1 Infection in Humanized Mice

Zhou, Jiehua, Neff, C Preston, Liu, Xiaoxuan, Zhang, Jane, Li, Haitang, Smith, David D, Swiderski, Piotr, Aboellail, Tawfik, Huang, Yuanyu, Du, Quan, Liang, Zicai, Peng, Ling, Akkina, Ramesh, Rossi, J

Journal:

Molecular Therapy

Année:

2011

Langue:

english

Fichier:

PDF, 466 KB

english, 2011

10

Rational Design of Therapeutic siRNAs: Minimizing Off-targeting Potential to Improve the Safety of RNAi Therapy for Huntington's Disease

Boudreau, Ryan L, Spengler, Ryan M, Davidson, Beverly L

Journal:

Molecular Therapy

Année:

2011

Langue:

english

Fichier:

PDF, 2.54 MB

english, 2011

11

Gene Therapy for SCID-X1: Focus on Clinical Data

Baum, Christopher

Journal:

Molecular Therapy

Année:

2011

Langue:

english

Fichier:

PDF, 205 KB

english, 2011

12

Efficient In Vivo Delivery of siRNA Into Brain Capillary Endothelial Cells Along With Endogenous Lipoprotein

Kuwahara, Hiroya, Nishina, Kazutaka, Yoshida, Kie, Nishina, Tomoko, Yamamoto, Mariko, Saito, Yukari, Piao, Wenying, Yoshida, Masayuki, Mizusawa, Hidehiro, Yokota, Takanori

Journal:

Molecular Therapy

Année:

2011

Langue:

english

Fichier:

PDF, 1.82 MB

english, 2011

13

Interfering With Tumor Pathways That Augment Viral Oncolysis

Journal:

Molecular Therapy

Année:

2011

Langue:

english

Fichier:

PDF, 219 KB

english, 2011

14

Gene Transfer Using HACs: A Key Step Closer to Ex Vivo Gene Therapy Using Autologous Gene–Corrected Cells to Treat Muscular Dystrophy

Tremblay, Jacques P, Frederickson, Robert M

Journal:

Molecular Therapy

Année:

2011

Langue:

english

Fichier:

PDF, 222 KB

english, 2011

15

Selective Therapeutic Targeting of the Anaplastic Lymphoma Kinase With Liposomal siRNA Induces Apoptosis and Inhibits Angiogenesis in Neuroblastoma

Di Paolo, Daniela, Ambrogio, Chiara, Pastorino, Fabio, Brignole, Chiara, Martinengo, Cinzia, Carosio, Roberta, Loi, Monica, Pagnan, Gabriella, Emionite, Laura, Cilli, Michele, Ribatti, Domenico, Allen

Journal:

Molecular Therapy

Année:

2011

Langue:

english

Fichier:

PDF, 2.86 MB

english, 2011

16

PiggyBac-mediated Cancer Immunotherapy Using EBV-specific Cytotoxic T-cells Expressing HER2-specific Chimeric Antigen Receptor

Nakazawa, Yozo, Huye, Leslie E, Salsman, Vita S, Leen, Ann M, Ahmed, Nabil, Rollins, Lisa, Dotti, Gianpietro, Gottschalk, Stephen M, Wilson, Matthew H, Rooney, Cliona M

Journal:

Molecular Therapy

Année:

2011

Langue:

english

Fichier:

PDF, 4.17 MB

english, 2011

17

Stabilization of Expanded (CTG)•(CAG) Repeats by Antisense Oligonucleotides

Nakamori, Masayuki, Gourdon, Geneviève, Thornton, Charles A

Journal:

Molecular Therapy

Année:

2011

Langue:

english

Fichier:

PDF, 589 KB

english, 2011

18

Microarray Analysis of Gene Expression Profiles in Cells Transfected With Nonviral Vectors

Plautz, Sarah A, Boanca, Gina, Riethoven, Jean-Jack M, Pannier, Angela K

Journal:

Molecular Therapy

Année:

2011

Langue:

english

Fichier:

PDF, 377 KB

english, 2011

19

Co-operative Membrane Disruption Between Cell-penetrating Peptide and Cargo: Implications for the Therapeutic Use of the Bcl-2 Converter Peptide D-NuBCP-9-r8

Watkins, Catherine L, Sayers, Edward J, Allender, Chris, Barrow, David, Fegan, Christopher, Brennan, Paul, Jones, Arwyn T

Journal:

Molecular Therapy

Année:

2011

Langue:

english

Fichier:

PDF, 2.22 MB

english, 2011

20

Phase Ia Clinical Evaluation of the Plasmodium falciparum Blood-stage Antigen MSP1 in ChAd63 and MVA Vaccine Vectors

Sheehy, Susanne H, Duncan, Christopher JA, Elias, Sean C, Collins, Katharine A, Ewer, Katie J, Spencer, Alexandra J, Williams, Andrew R, Halstead, Fenella D, Moretz, Samuel E, Miura, Kazutoyo, Epp, Ch

Journal:

Molecular Therapy

Année:

2011

Langue:

english

Fichier:

PDF, 1.67 MB

english, 2011

21

Combining mTor Inhibitors With Rapamycin-resistant T Cells: A Two-pronged Approach to Tumor Elimination

Huye, Leslie E, Nakazawa, Yozo, Patel, Mayuri P, Yvon, Eric, Sun, Jiali, Savoldo, Barbara, Wilson, Matthew H, Dotti, Gianpietro, Rooney, Cliona M

Journal:

Molecular Therapy

Année:

2011

Langue:

english

Fichier:

PDF, 915 KB

english, 2011

22

In This Issue

Journal:

Molecular Therapy

Année:

2011

Langue:

english

Fichier:

PDF, 1.39 MB

english, 2011

23

Research Highlights

Journal:

Molecular Therapy

Année:

2011

Langue:

english

Fichier:

PDF, 736 KB

english, 2011