Pulmonary Targeting of Adeno-associated Viral Vectors by Next-generation Sequencing-guided Screening of Random Capsid Displayed Peptide Libraries
52;rgen A Kleinschmidt & Martin Trepel (Source: Molecular Therapy)
Source: Molecular Therapy - June 20, 2016 Category: Genetics & Stem Cells Authors: Jakob KörbelinTimo SieberStefan MichelfelderLars LundingElmar SpiesAgnes HungerMalik AlawiKleopatra RaptiDaniela IndenbirkenOliver J MüllerRenata PasqualiniWadih ArapJürgen A KleinschmidtMartin Trepel Source Type: research
Superior In vivo Transduction of Human Hepatocytes Using Engineered AAV3 Capsid
Jong & Roland W Herzog (Source: Molecular Therapy)
Source: Molecular Therapy - June 20, 2016 Category: Genetics & Stem Cells Authors: Koen VercauterenBrad E HoffmanIrene ZolotukhinGeoffrey D KeelerJing W XiaoEtiena Basner-TschakarjanKatherine A HighHildegund CJ ErtlCharles M RiceArun SrivastavaYpe P de JongRoland W Herzog Source Type: research
rAAV Gene Therapy in a Canavan’s Disease Mouse Model Reveals Immune Impairments and an Extended Pathology Beyond the Central Nervous System
rAAV Gene Therapy in a Canavan’s Disease Mouse Model Reveals Immune Impairments and an Extended Pathology Beyond the Central Nervous System
Molecular Therapy 24,
1030 (June 2016). doi:10.1038/mt.2016.68
Authors: Seemin Seher Ahmed, Stefan A Schattgen, Ashley E Frakes, Elif M Sikoglu, Qin Su, Jia Li, Thomas G Hampton, Andrew R Denninger, Daniel A Kirschner, Brian Kaspar, Reuben Matalon & Guangping Gao (Source: Molecular Therapy)
Source: Molecular Therapy - June 20, 2016 Category: Genetics & Stem Cells Authors: Seemin Seher AhmedStefan A SchattgenAshley E FrakesElif M SikogluQin SuJia LiThomas G HamptonAndrew R DenningerDaniel A KirschnerBrian KasparReuben MatalonGuangping Gao Source Type: research
CNS-accessible Inhibitor of Glucosylceramide Synthase for Substrate Reduction Therapy of Neuronopathic Gaucher Disease
Leonard & Seng H Cheng (Source: Molecular Therapy)
Source: Molecular Therapy - June 20, 2016 Category: Genetics & Stem Cells Authors: John MarshallYing SunDinesh S BangariEva BudmanHyejung ParkJennifer B NietupskiAmy AllaireMary A CromwellBing WangGregory A GrabowskiJohn P LeonardSeng H Cheng Source Type: research
Timing of Gene Therapy Interventions: The Earlier, the Better
Molecular Therapy 24,
1017 (June 2016). doi:10.1038/mt.2016.20
Author: Steven J Gray (Source: Molecular Therapy)
Source: Molecular Therapy - June 20, 2016 Category: Genetics & Stem Cells Authors: Steven J Gray Source Type: research
Research Highlights
Molecular Therapy 24,
1016 (June 2016). doi:10.1038/mt.2016.100 (Source: Molecular Therapy)
Source: Molecular Therapy - June 20, 2016 Category: Genetics & Stem Cells Source Type: research
In This Issue
Molecular Therapy 24,
1015 (June 2016). doi:10.1038/mt.2016.99 (Source: Molecular Therapy)
Source: Molecular Therapy - June 20, 2016 Category: Genetics & Stem Cells Source Type: research
ADA-SCID Gene Therapy Endorsed By European Medicines Agency For Marketing Authorization
Molecular Therapy 24,
1013 (June 2016). doi:10.1038/mt.2016.98
Author: Seppo Ylä-Herttuala (Source: Molecular Therapy)
Source: Molecular Therapy - June 20, 2016 Category: Genetics & Stem Cells Authors: Seppo Ylä-Herttuala Source Type: research
Development of an Interleukin-1β Vaccine in Patients with Type 2 Diabetes
Development of an Interleukin-1β Vaccine in Patients with Type 2 Diabetes
Molecular Therapy 24,
1003 (May 2016). doi:10.1038/mt.2015.227
Authors: Claudia Cavelti-Weder, Katharina Timper, Eleonora Seelig, Cornelia Keller, Martin Osranek, Ute Lässing, Gunther Spohn, Patrik Maurer, Philipp Müller, Gary T Jennings, Joerg Willers, Philippe Saudan, Marc Y Donath & Martin F Bachmann (Source: Molecular Therapy)
Source: Molecular Therapy - May 19, 2016 Category: Genetics & Stem Cells Authors: Claudia Cavelti-WederKatharina TimperEleonora SeeligCornelia KellerMartin OsranekUte LässingGunther SpohnPatrik MaurerPhilipp MüllerGary T JenningsJoerg WillersPhilippe SaudanMarc Y DonathMartin F Bachmann Source Type: research
H7N9 Live Attenuated Influenza Vaccine Is Highly Immunogenic, Prevents Virus Replication, and Protects Against Severe Bronchopneumonia in Ferrets
oll & Larisa Rudenko (Source: Molecular Therapy)
Source: Molecular Therapy - May 19, 2016 Category: Genetics & Stem Cells Authors: Jørgen de JongeIrina Isakova-SivakHarry van DijkenSanne SpijkersJustin MouthaanRineke de JongTatiana SmolonoginaPaul RohollLarisa Rudenko Source Type: research
A Role for the Long Noncoding RNA SENCR in Commitment and Function of Endothelial Cells
C Mountford & Andrew H Baker (Source: Molecular Therapy)
Source: Molecular Therapy - May 19, 2016 Category: Genetics & Stem Cells Authors: Mounia BoulberdaaElizabeth ScottMargaret BallantyneRaquel GarciaBetty DescampsGianni D AngeliniMairi BrittanAmanda HunterMartin McBrideJohn McClureJoseph M MianoCostanza EmanueliNicholas L MillsJoanne C MountfordAndrew H Baker Source Type: research
Human Mesenchymal Stem Cells Genetically Engineered to Overexpress Brain-derived Neurotrophic Factor Improve Outcomes in Huntington’s Disease Mouse Models
Human Mesenchymal Stem Cells Genetically Engineered to Overexpress Brain-derived Neurotrophic Factor Improve Outcomes in Huntington’s Disease Mouse Models
Molecular Therapy 24,
965 (May 2016). doi:10.1038/mt.2016.12
Authors: Kari Pollock, Heather Dahlenburg, Haley Nelson, Kyle D Fink, Whitney Cary, Kyle Hendrix, Geralyn Annett, Audrey Torrest, Peter Deng, Joshua Gutierrez, Catherine Nacey, Karen Pepper, Stefanos Kalomoiris, Johnathon D Anderson, Jeannine McGee, William Gruenloh, Brian Fury, Gerhard Bauer, Alexandria Duffy, Theresa Tempkin, Vicki Wheelock & Jan A Nolta (Source: Molecular Therapy)
Source: Molecular Therapy - May 19, 2016 Category: Genetics & Stem Cells Authors: Kari PollockHeather DahlenburgHaley NelsonKyle D FinkWhitney CaryKyle HendrixGeralyn AnnettAudrey TorrestPeter DengJoshua GutierrezCatherine NaceyKaren PepperStefanos KalomoirisJohnathon D AndersonJeannine McGeeWilliam GruenlohBrian FuryGerhard BauerAlexa Source Type: research
Induced Apoptosis Investigation in Wild-type and FLT3-ITD Acute Myeloid Leukemia Cells by Nanochannel Electroporation and Single-cell qRT-PCR
e & Ly James Lee (Source: Molecular Therapy)
Source: Molecular Therapy - May 19, 2016 Category: Genetics & Stem Cells Authors: Keliang GaoXiaomeng HuangChi-Ling ChiangXinmei WangLingqian ChangPouyan BoukanyGuido MarcucciRobert LeeLy James Lee Source Type: research
Argonaute 2-dependent Regulation of Gene Expression by Single-stranded miRNA Mimics
R Corey (Source: Molecular Therapy)
Source: Molecular Therapy - May 19, 2016 Category: Genetics & Stem Cells Authors: Masayuki MatsuiThazha P PrakashDavid R Corey Source Type: research
MiR-298 Counteracts Mutant Androgen Receptor Toxicity in Spinal and Bulbar Muscular Atrophy
ck & Carlo Rinaldi (Source: Molecular Therapy)
Source: Molecular Therapy - May 19, 2016 Category: Genetics & Stem Cells Authors: Naemeh PourshafiePhilip R LeeKe-lian ChenGeorge G HarmisonLaura C BottMasahisa KatsunoGen SobueBarrington G BurnettKenneth H FischbeckCarlo Rinaldi Source Type: research
