Glycolate Oxidase Is a Safe and Efficient Target for Substrate Reduction Therapy in a Mouse Model of Primary Hyperoxaluria Type I
o Salido (Source: Molecular Therapy)
Source: Molecular Therapy - April 14, 2016 Category: Genetics & Stem Cells Authors: Cristina Martin-HiguerasSergio Luis-LimaEduardo Salido Source Type: research
Modulation of Protein–Protein Interactions for the Development of Novel Therapeutics
Modulation of Protein–Protein Interactions for the Development of Novel Therapeutics
Molecular Therapy 24,
707 (April 2016). doi:10.1038/mt.2015.214
Authors: Ioanna Petta, Sam Lievens, Claude Libert, Jan Tavernier & Karolien De Bosscher (Source: Molecular Therapy)
Source: Molecular Therapy - April 14, 2016 Category: Genetics & Stem Cells Authors: Ioanna PettaSam LievensClaude LibertJan TavernierKarolien De Bosscher Source Type: research
In Vivo Zinc Finger Nuclease-mediated Targeted Integration of a Glucose-6-phosphatase Transgene Promotes Survival in Mice With Glycogen Storage Disease Type IA
mp; Dwight D Koeberl (Source: Molecular Therapy)
Source: Molecular Therapy - April 14, 2016 Category: Genetics & Stem Cells Authors: Dustin J LandauElizabeth Drake BrooksPablo Perez-PineraHiruni AmarasekaraAdam MefferdSongtao LiAndrew BirdCharles A GersbachDwight D Koeberl Source Type: research
Precise Correction of Disease Mutations in Induced Pluripotent Stem Cells Derived From Patients With Limb Girdle Muscular Dystrophy
hele P Calos (Source: Molecular Therapy)
Source: Molecular Therapy - April 14, 2016 Category: Genetics & Stem Cells Authors: Soeren TuranAlfonso P FarruggioWaracharee SrifaJohn W DayMichele P Calos Source Type: research
Gene Insertion Into Genomic Safe Harbors for Human Gene Therapy
ambach (Source: Molecular Therapy)
Source: Molecular Therapy - April 14, 2016 Category: Genetics & Stem Cells Authors: Eirini P PapapetrouAxel Schambach Source Type: research
Progress With Developing Use of Gene Editing To Cure Chronic Infection With Hepatitis B Virus
rbuthnot (Source: Molecular Therapy)
Source: Molecular Therapy - April 14, 2016 Category: Genetics & Stem Cells Authors: Abdullah ElyBuhle MoyoPatrick Arbuthnot Source Type: research
Gene Therapy for Hemoglobinopathies: Tremendous Successes and Remaining Caveats
Molecular Therapy 24,
668 (April 2016). doi:10.1038/mt.2016.57
Author: Punam Malik (Source: Molecular Therapy)
Source: Molecular Therapy - April 14, 2016 Category: Genetics & Stem Cells Authors: Punam Malik Source Type: research
siRNA Therapeutics for Primary Hyperoxaluria: A Beginning
Molecular Therapy 24,
666 (April 2016). doi:10.1038/mt.2016.50
Author: Dawn S Milliner (Source: Molecular Therapy)
Source: Molecular Therapy - April 14, 2016 Category: Genetics & Stem Cells Authors: Dawn S Milliner Source Type: research
AAVR: A Multi-Serotype Receptor for AAV
mulski (Source: Molecular Therapy)
Source: Molecular Therapy - April 14, 2016 Category: Genetics & Stem Cells Authors: Candace SummerfordR. Jude Samulski Source Type: research
Reply to “Wild-type AAV Insertions in Hepatocellular Carcinoma Do Not Inform Debate Over Genotoxicity Risk of Vectorized AAV”
Reply to “Wild-type AAV Insertions in Hepatocellular Carcinoma Do Not Inform Debate Over Genotoxicity Risk of Vectorized AAV”
Molecular Therapy 24,
661 (April 2016). doi:10.1038/mt.2016.48
Authors: Manfred Schmidt, Irene Gil-Farina & Hildegard Büning (Source: Molecular Therapy)
Source: Molecular Therapy - April 14, 2016 Category: Genetics & Stem Cells Authors: Manfred SchmidtIrene Gil-FarinaHildegard Büning Source Type: research
Wild-type AAV Insertions in Hepatocellular Carcinoma Do Not Inform Debate Over Genotoxicity Risk of Vectorized AAV
s Laurent, Mélanie Letexier, Paulette Bioulac-Sage, Fabien Calvo & Jessica Zucman-Rossi (Source: Molecular Therapy)
Source: Molecular Therapy - April 14, 2016 Category: Genetics & Stem Cells Authors: Jean-Charles NaultIadh MamiTiziana La BellaShalini DattaSandrine ImbeaudAndrea FranconiMaxime MalletGabrielle CouchyEric LetouzéCamilla PilatiBenjamin VerretJean-Frédéric BlancCharles BalabaudJulien CalderaroAlexis LaurentMélanie LetexierPaulette Biou Source Type: research
Research Highlights
Molecular Therapy 24,
659 (April 2016). doi:10.1038/mt.2016.46 (Source: Molecular Therapy)
Source: Molecular Therapy - April 14, 2016 Category: Genetics & Stem Cells Source Type: research
In This Issue
Molecular Therapy 24,
658 (April 2016). doi:10.1038/mt.2016.45 (Source: Molecular Therapy)
Source: Molecular Therapy - April 14, 2016 Category: Genetics & Stem Cells Source Type: research
Expanded Opportunities for Methods and Clinical Development
Molecular Therapy 24,
655 (April 2016). doi:10.1038/mt.2016.44
Author: Roland W Herzog (Source: Molecular Therapy)
Source: Molecular Therapy - April 14, 2016 Category: Genetics & Stem Cells Authors: Roland W Herzog Source Type: research
The Neisseria meningitidis CRISPR-Cas9 System Enables Specific Genome Editing in Mammalian Cells
Gang Bao (Source: Molecular Therapy)
Source: Molecular Therapy - March 8, 2016 Category: Genetics & Stem Cells Authors: Ciaran M LeeThomas J CradickGang Bao Source Type: research
