Streptococcus thermophilus CRISPR-Cas9 Systems Enable Specific Editing of the Human Genome
& Claudio Mussolino (Source: Molecular Therapy)
Source: Molecular Therapy - March 8, 2016 Category: Genetics & Stem Cells Authors: Maximilian MüllerCiaran M LeeGiedrius GasiunasTimothy H DavisThomas J CradickVirginijus SiksnysGang BaoToni CathomenClaudio Mussolino Source Type: research
Sleeping Beauty Transposon Vectors in Liver-directed Gene Delivery of LDLR and VLDLR for Gene Therapy of Familial Hypercholesterolemia
mp; Seppo Ylä-Herttuala (Source: Molecular Therapy)
Source: Molecular Therapy - March 8, 2016 Category: Genetics & Stem Cells Authors: Tytteli A K TurunenJere KurkipuroTommi HeikuraTaina VuorioElisa HytönenZsuzsanna IzsvákSeppo Ylä-Herttuala Source Type: research
Multidimensional Genome-wide Analyses Show Accurate FVIII Integration by ZFN in Primary Human Cells
troh & Oi Lian Kon (Source: Molecular Therapy)
Source: Molecular Therapy - March 8, 2016 Category: Genetics & Stem Cells Authors: Jaichandran SivalingamDimitar KenanovHao HanAjit Johnson NirmalWai Har NgSze Sing LeeJeyakumar MasilamaniToan Thang PhanSebastian Maurer-StrohOi Lian Kon Source Type: research
Genome-wide Profiling Reveals Remarkable Parallels Between Insertion Site Selection Properties of the MLV Retrovirus and the piggyBac Transposon in Primary Human CD4+ T Cells
zsvák & Zoltán Ivics (Source: Molecular Therapy)
Source: Molecular Therapy - March 8, 2016 Category: Genetics & Stem Cells Authors: Andreas Gogol-DöringIsmahen AmmarSaumyashree GuptaMario BunseCsaba MiskeyWei ChenWolfgang UckertThomas F SchulzZsuzsanna IzsvákZoltán Ivics Source Type: research
Silent IL2RG Gene Editing in Human Pluripotent Stem Cells
p; David W Russell (Source: Molecular Therapy)
Source: Molecular Therapy - March 8, 2016 Category: Genetics & Stem Cells Authors: Li B LiChao MaGeneve AwongMarion KennedyGerman GornalusseGordon KellerDan S KaufmanDavid W Russell Source Type: research
Evaluation of TCR Gene Editing Achieved by TALENs, CRISPR/Cas9, and megaTAL Nucleases
anfred Schmidt, Richard Gabriel, Keli L Hippen, Jeffrey S Miller, Andrew M Scharenberg, Jakub Tolar & Bruce R Blazar (Source: Molecular Therapy)
Source: Molecular Therapy - March 8, 2016 Category: Genetics & Stem Cells Authors: Mark J OsbornBeau R WebberFriederike KnippingCara-lin LonetreeNicole TennisAnthony P DeFeoAmber N McElroyColby G StarkerCatherine LeeSarah MerkelTroy C LundKaren S Kelly-SprattMichael C JensenDaniel F VoytasChristof von KalleManfred SchmidtRichard Gabriel Source Type: research
CRISPR-mediated Genome Editing Restores Dystrophin Expression and Function in mdx Mice
Ma & Renzhi Han (Source: Molecular Therapy)
Source: Molecular Therapy - March 8, 2016 Category: Genetics & Stem Cells Authors: Li XuKi Ho ParkLixia ZhaoJing XuMona El RefaeyYandi GaoHua ZhuJianjie MaRenzhi Han Source Type: research
In Vivo CRISPR/Cas9 Gene Editing Corrects Retinal Dystrophy in the S334ter-3 Rat Model of Autosomal Dominant Retinitis Pigmentosa
ndsen & Shaomei Wang (Source: Molecular Therapy)
Source: Molecular Therapy - March 8, 2016 Category: Genetics & Stem Cells Authors: Benjamin BakondiWenjian LvBin LuMelissa K JonesYuchun TsaiKevin J KimRachelle LevyAslam Abbasi AkhtarJoshua J BreunigClive N SvendsenShaomei Wang Source Type: research
Protein Delivery of an Artificial Transcription Factor Restores Widespread Ube3a Expression in an Angelman Syndrome Mouse Brain
an & David J Segal (Source: Molecular Therapy)
Source: Molecular Therapy - March 8, 2016 Category: Genetics & Stem Cells Authors: Barbara J BailusBenjamin PylesMichelle M McAlisterHenriette O'GeenSarah H LockwoodAlexa N AdamsJennifer Trang T NguyenAbigail YuRobert F BermanDavid J Segal Source Type: research
Re-expression of Selected Epigenetically Silenced Candidate Tumor Suppressor Genes in Cervical Cancer by TET2-directed Demethylation
& Marianne G Rots (Source: Molecular Therapy)
Source: Molecular Therapy - March 8, 2016 Category: Genetics & Stem Cells Authors: Christian HuismanMonique G P van der WijstMatthijs SchokkerPilar BlancafortMartijn M TerpstraKlaas KokAte G J van der ZeeEd SchuuringG Bea A WismanMarianne G Rots Source Type: research
CRISPR/dCas9-mediated Transcriptional Inhibition Ameliorates the Epigenetic Dysregulation at D4Z4 and Represses DUX4-fl in FSH Muscular Dystrophy
L Jones (Source: Molecular Therapy)
Source: Molecular Therapy - March 8, 2016 Category: Genetics & Stem Cells Authors: Charis L HimedaTakako I JonesPeter L Jones Source Type: research
CRISPR-Cas9 Can Inhibit HIV-1 Replication but NHEJ Repair Facilitates Virus Escape
Atze T Das (Source: Molecular Therapy)
Source: Molecular Therapy - March 8, 2016 Category: Genetics & Stem Cells Authors: Gang WangNa ZhaoBen BerkhoutAtze T Das Source Type: research
Specific Reactivation of Latent HIV-1 by dCas9-SunTag-VP64-mediated Guide RNA Targeting the HIV-1 Promoter
hua Wang & Huanzhang Zhu (Source: Molecular Therapy)
Source: Molecular Therapy - March 8, 2016 Category: Genetics & Stem Cells Authors: Haiyan JiZhengtao JiangPanpan LuLi MaChuan LiHanyu PanZheng FuXiying QuPengfei WangJunxiao DengXinyi YangJianhua WangHuanzhang Zhu Source Type: research
CRISPR-mediated Activation of Latent HIV-1 Expression
Schaffer (Source: Molecular Therapy)
Source: Molecular Therapy - March 8, 2016 Category: Genetics & Stem Cells Authors: Prajit LimsirichaiThomas GajDavid V Schaffer Source Type: research
Potent and Targeted Activation of Latent HIV-1 Using the CRISPR/dCas9 Activator Complex
amp; Marc S Weinberg (Source: Molecular Therapy)
Source: Molecular Therapy - March 8, 2016 Category: Genetics & Stem Cells Authors: Sheena M SaaymanDaniel C LazarTristan A ScottJonathan R HartMayumi TakahashiJohn C BurnettVicente PlanellesKevin V MorrisMarc S Weinberg Source Type: research
