Citations (121)
Keep up to date with the latest research on this topic with citation updates for this article.
Read on this site (5)
Yanqing Li, Sai Li, Yan Li, Haibin Xia & Qinwen Mao. (2019) Generation of a novel HEK293 luciferase reporter cell line by CRISPR/Cas9-mediated site-specific integration in the genome to explore the transcriptional regulation of the PGRN gene. Bioengineered 10:1, pages 98-107.
Read now
Read now
Gurbachan S. Miglani. (2017) Genome editing in crop improvement: Present scenario and future prospects. Journal of Crop Improvement 31:4, pages 453-559.
Read now
Read now
Olivier Humbert, Luther Davis & Nancy Maizels. (2012) Targeted gene therapies: tools, applications, optimization. Critical Reviews in Biochemistry and Molecular Biology 47:3, pages 264-281.
Read now
Read now
Marlon R. Veldwijk, Leopold Sellner, Marius Stiefelhagen, Jürgen A. Kleinschmidt, Stephanie Laufs, Julian Topaly, Stefan Fruehauf, W. Jens Zeller & Frederik Wenz. (2010) Pseudotyped recombinant adeno-associated viral vectors mediate efficient gene transfer into primary human CD34+ peripheral blood progenitor cells. Cytotherapy 12:1, pages 107-112.
Read now
Read now
Matthew Porteus. (2007) Using Homologous Recombination to Manipulate the Genome of Human Somatic Cells. Biotechnology and Genetic Engineering Reviews 24:1, pages 195-212.
Read now
Read now
Articles from other publishers (116)
Mara Pavel-Dinu, Simon Borna & Rosa Bacchetta. (2023) Rare immune diseases paving the road for genome editing-based precision medicine. Frontiers in Genome Editing 5.
Crossref
Crossref
Shinnosuke Tsuji, Calvin J. Stephens, Giulia Bortolussi, Feijie Zhang, Gabriele Baj, Hagoon Jang, Gustavo de Alencastro, Andrés F. Muro, Katja Pekrun & Mark A. Kay. (2022) Fludarabine increases nuclease-free AAV- and CRISPR/Cas9-mediated homologous recombination in mice. Nature Biotechnology 40:8, pages 1285-1294.
Crossref
Crossref
Myriam L.R. Haltalli, Adam C. Wilkinson, Alejo Rodriguez-Fraticelli & Matthew Porteus. (2022) Hematopoietic stem cell gene editing and expansion: State-of-the-art technologies and recent applications. Experimental Hematology 107, pages 9-13.
Crossref
Crossref
Swati Bijlani, Ka Ming Pang, Venkatesh Sivanandam, Amanpreet Singh & Saswati Chatterjee. (2022) The Role of Recombinant AAV in Precise Genome Editing. Frontiers in Genome Editing 3.
Crossref
Crossref
Takayasu Mikuni & Motokazu Uchigashima. (2020) Methodological approaches to understand the molecular mechanism of structural plasticity of dendritic spines. European Journal of Neuroscience 54:8, pages 6902-6911.
Crossref
Crossref
Sriram Vaidyanathan, Matthew McCarra & Tushar J. Desai. 2021. Lung Stem Cells in Development, Health and Disease. Lung Stem Cells in Development, Health and Disease
306
321
.
Laura P. Spector, Matthew Tiffany, Nicole M. Ferraro, Nathan S. Abell, Stephen B. Montgomery & Mark A. Kay. (2021) Evaluating the Genomic Parameters Governing rAAV-Mediated Homologous Recombination. Molecular Therapy 29:3, pages 1028-1046.
Crossref
Crossref
Hidde A. Zittersteijn, Manuel A.F.V. Gonçalves & Rob C. Hoeben. (2020) A primer to gene therapy: Progress, prospects, and problems. Journal of Inherited Metabolic Disease 44:1, pages 54-71.
Crossref
Crossref
Huei-Mei Chen, Rachel Resendes, Azita Ghodssi, Danielle Sookiasian, Michael Tian, Serena Dollive, Laura Adamson-Small, Nancy Avila, Cagdas Tazearslan, John F. Thompson, Jeff L. Ellsworth, Omar Francone, Albert Seymour & Jason B. Wright. (2020) Molecular characterization of precise in vivo targeted gene integration in human cells using AAVHSC15. PLOS ONE 15:5, pages e0233373.
Crossref
Crossref
Anna C. Maurer & Matthew D. Weitzman. (2020) Adeno-Associated Virus Genome Interactions Important for Vector Production and Transduction. Human Gene Therapy 31:9-10, pages 499-511.
Crossref
Crossref
Saswati ChatterjeeVenkatesh SivanandamKamehameha Kai-Min Wong, Jr., Jr.. (2020) Adeno-Associated Virus and Hematopoietic Stem Cells: The Potential of Adeno-Associated Virus Hematopoietic Stem Cells in Genetic Medicines. Human Gene Therapy 31:9-10, pages 542-552.
Crossref
Crossref
Marcella Brescia, Josephine M. Janssen, Jin Liu & Manuel A. F. V. Gonçalves. (2020) High-Capacity Adenoviral Vectors Permit Robust and Versatile Testing of DMD Gene Repair Tools and Strategies in Human Cells. Cells 9:4, pages 869.
Crossref
Crossref
Takayasu Mikuni. (2020) Genome editing-based approaches for imaging protein localization and dynamics in the mammalian brain. Neuroscience Research 150, pages 2-7.
Crossref
Crossref
Pablo D. Moço, Noga Aharony & Amine Kamen. (2019) Adeno‐Associated Viral Vectors for Homology‐Directed Generation of CAR‐T Cells. Biotechnology Journal 15:1, pages 1900286.
Crossref
Crossref
Amanda M. Dudek & Matthew H. Porteus. (2019) AAV6 Is Superior to Clade F AAVs in Stimulating Homologous Recombination-Based Genome Editing in Human HSPCs. Molecular Therapy 27:10, pages 1701-1705.
Crossref
Crossref
Mara Pavel-Dinu, Volker Wiebking, Beruh T. Dejene, Waracharee Srifa, Sruthi Mantri, Carmencita E. Nicolas, Ciaran Lee, Gang Bao, Eric J. Kildebeck, Niraj Punjya, Camille Sindhu, Matthew A. Inlay, Nivedita Saxena, Suk See DeRavin, Harry Malech, Maria Grazia Roncarolo, Kenneth I. Weinberg & Matthew H. Porteus. (2019) Gene correction for SCID-X1 in long-term hematopoietic stem cells. Nature Communications 10:1.
Crossref
Crossref
Jun Nishiyama. (2019) Genome editing in the mammalian brain using the CRISPR–Cas system. Neuroscience Research 141, pages 4-12.
Crossref
Crossref
M. Kyle Cromer, Sriram Vaidyanathan, Daniel E. Ryan, Bo Curry, Anne Bergstrom Lucas, Joab Camarena, Milan Kaushik, Sarah R. Hay, Renata M. Martin, Israel Steinfeld, Rasmus O. Bak, Daniel P. Dever, Ayal Hendel, Laurakay Bruhn & Matthew H. Porteus. (2018) Global Transcriptional Response to CRISPR/Cas9-AAV6-Based Genome Editing in CD34+ Hematopoietic Stem and Progenitor Cells. Molecular Therapy 26:10, pages 2431-2442.
Crossref
Crossref
Beatrice Ho, Sharon Loh, Woon Chan & Boon Soh. (2018) In Vivo Genome Editing as a Therapeutic Approach. International Journal of Molecular Sciences 19:9, pages 2721.
Crossref
Crossref
Michal Alon, Rafi Emmanuel, Nouar Qutob, Anna Bakhman, Victoria Peshti, Alexandra Brodezki, David Bassan, Mickey Kosloff & Yardena Samuels. (2018)
Refinement of the endogenous epitope tagging technology allows the identification of a novel
NRAS
binding partner in melanoma
. Pigment Cell & Melanoma Research 31:5, pages 641-648.
Crossref
Crossref
Laura J. SmithJason WrightGabriella ClarkTaihra Ul-HasanXiangyang JinAbigail FongManasa ChandraThia St MartinHillard RubinDavid KnowltonJeff L. EllsworthYuman FongKamehameha K. WongJr.Jr. & Saswati Chatterjee. (2018) Stem cell-derived clade F AAVs mediate high-efficiency homologous recombination-based genome editing. Proceedings of the National Academy of Sciences 115:31.
Crossref
Crossref
Jun Nishiyama, Takayasu Mikuni & Ryohei Yasuda. (2017) Virus-Mediated Genome Editing via Homology-Directed Repair in Mitotic and Postmitotic Cells in Mammalian Brain. Neuron 96:4, pages 755-768.e5.
Crossref
Crossref
Michael C. Gundry, Daniel P. Dever, David Yudovich, Daniel E. Bauer, Simon Haas, Adam C. Wilkinson & Sofie Singbrant. (2017) Technical considerations for the use of CRISPR/Cas9 in hematology research. Experimental Hematology 54, pages 4-11.
Crossref
Crossref
Yuxia LuoAmy FrederickJohn M. MartinAbraham ScariaSeng H. ChengDonna ArmentanoSamuel C. WadsworthKaren A. Vincent. (2017) AAVS1-Targeted Plasmid Integration in AAV Producer Cell Lines. Human Gene Therapy Methods 28:3, pages 124-138.
Crossref
Crossref
Christopher E. Nelson & Charles A. Gersbach. (2016) Engineering Delivery Vehicles for Genome Editing. Annual Review of Chemical and Biomolecular Engineering 7:1, pages 637-662.
Crossref
Crossref
Sivasundaram Karnan, Akinobu Ota, Yuko Konishi, Md Wahiduzzaman, Yoshitaka Hosokawa & Hiroyuki Konishi. (2016) Improved methods of AAV-mediated gene targeting for human cell lines using ribosome-skipping 2A peptide. Nucleic Acids Research 44:6, pages e54-e54.
Crossref
Crossref
Morgan L Maeder & Charles A Gersbach. (2016) Genome-editing Technologies for Gene and Cell Therapy. Molecular Therapy 24:3, pages 430-446.
Crossref
Crossref
Li B Li, Chao Ma, Geneve Awong, Marion Kennedy, German Gornalusse, Gordon Keller, Dan S Kaufman & David W Russell. (2016) Silent IL2RG Gene Editing in Human Pluripotent Stem Cells. Molecular Therapy 24:3, pages 582-591.
Crossref
Crossref
Xiaoyu Chen & Manuel A F V Gonçalves. (2016) Engineered Viruses as Genome Editing Devices. Molecular Therapy 24:3, pages 447-457.
Crossref
Crossref
Thomas Gaj, Benjamin E Epstein & David V Schaffer. (2016) Genome Engineering Using Adeno-associated Virus: Basic and Clinical Research Applications. Molecular Therapy 24:3, pages 458-464.
Crossref
Crossref
Matthew Porteus. (2016) Genome Editing: A New Approach to Human Therapeutics. Annual Review of Pharmacology and Toxicology 56:1, pages 163-190.
Crossref
Crossref
Ruth R. Shah, Justyna Cholewa-Waclaw, Faith C.J. Davies, Katie M. Paton, Ronan Chaligne, Edith Heard, Catherine M. Abbott & Adrian P. Bird. (2016) Efficient and versatile CRISPR engineering of human neurons in culture to model neurological disorders. Wellcome Open Research 1, pages 13.
Crossref
Crossref
Cristina Chamorro, Angeles Mencía, David Almarza, Blanca Duarte, Hildegard Büning, Jessica Sallach, Ingrid Hausser, Marcela Del Río, Fernando Larcher & Rodolfo Murillas. (2016) Gene Editing for the Efficient Correction of a Recurrent COL7A1 Mutation in Recessive Dystrophic Epidermolysis Bullosa Keratinocytes. Molecular Therapy - Nucleic Acids 5, pages e307.
Crossref
Crossref
David G. Ousterout & Charles A. Gersbach. 2016. Genome Editing. Genome Editing
51
79
.
Giel Vandemoortele, Kris Gevaert & Sven Eyckerman. (2016) Proteomics in the genome engineering era. PROTEOMICS 16:2, pages 177-187.
Crossref
Crossref
Matthew H. Porteus. (2015) Towards a new era in medicine: therapeutic genome editing. Genome Biology 16:1.
Crossref
Crossref
Rajiv Sharma, Xavier M. Anguela, Yannick Doyon, Thomas Wechsler, Russell C. DeKelver, Scott Sproul, David E. Paschon, Jeffrey C. Miller, Robert J. DavidsonDavid Shivak, Shangzhen ZhouJulianne RiedersPhilip D. Gregory, Michael C. Holmes, Edward J. Rebar & Katherine A. High. (2015) In vivo genome editing of the albumin locus as a platform for protein replacement therapy. Blood 126:15, pages 1777-1784.
Crossref
Crossref
Blythe D. SatherGuillermo S. Romano IbarraKaren SommerGabrielle CuringaMalika HaleIram F. KhanSwati SinghYumei SongKamila GwiazdaJaya SahniJordan JarjourAlexander AstrakhanThor A. WagnerAndrew M. ScharenbergDavid J. Rawlings. (2015)
Efficient modification of
CCR5
in primary human hematopoietic cells using a megaTAL nuclease and AAV donor template
. Science Translational Medicine 7:307.
Crossref
Crossref
M L Hirsch. (2014) Adeno-associated virus inverted terminal repeats stimulate gene editing. Gene Therapy 22:2, pages 190-195.
Crossref
Crossref
David R Deyle, R Scott Hansen, Anda M Cornea, Li B Li, Amber A Burt, Ian E Alexander, Richard S Sandstrom, John A Stamatoyannopoulos, Chia-Lin Wei & David W Russell. (2014) A genome-wide map of adeno-associated virus–mediated human gene targeting. Nature Structural & Molecular Biology 21:11, pages 969-975.
Crossref
Crossref
H-H Zhang, S-Z Li, Z-Y Zhang, X-M Hu, P-N Hou, L Gao, R-L Du & X-D Zhang. (2014) Nemo-like kinase is critical for p53 stabilization and function in response to DNA damage. Cell Death & Differentiation 21:10, pages 1656-1663.
Crossref
Crossref
Wonkyun Choi, Beom-Ho Jo, Min-A Seol, Soon-Jae Eum, Jun Hyung Park & Hae-Ryong Song. (2014) Presence of Environmental Risk Assessments for LMOs in nature and Future Considerations based on New Biotechnologies. Journal of the Korean Society of International Agricultue 26:3, pages 297-302.
Crossref
Crossref
Carmen Bertoni. (2014) Emerging gene editing strategies for Duchenne muscular dystrophy targeting stem cells. Frontiers in Physiology 5.
Crossref
Crossref
David R. Deyle, Li B. Li, Gaoying Ren & David W. Russell. (2014) The effects of polymorphisms on human gene targeting. Nucleic Acids Research 42:5, pages 3119-3124.
Crossref
Crossref
H M Elbadawy, M Gailledrat, C Desseaux, G Salvalaio, E Di Iorio, B Ferrari, M Bertolin, V Barbaro, M Parekh, R Gayon, D Munegato, E Franchin, A Calistri, G Palù, C Parolin, D Ponzin & S Ferrari. (2014) Gene transfer of integration defective anti-HSV-1 meganuclease to human corneas ex vivo. Gene Therapy 21:3, pages 272-281.
Crossref
Crossref
Katia Pauwels, Nancy Podevin, Didier Breyer, Dana Carroll & Philippe Herman. (2014) Engineering nucleases for gene targeting: safety and regulatory considerations. New Biotechnology 31:1, pages 18-27.
Crossref
Crossref
Xia Zhang & Jorge A. Piedrahita. 2014. Principles of Cloning. Principles of Cloning
95
106
.
Matthew L. Hirsch & R. Jude Samulski. 2014. Gene Correction. Gene Correction
291
307
.
Brian L Ellis, Matthew L Hirsch, Jenny C Barker, Jon P Connelly, Robert J SteiningerIIIIII & Matthew H Porteus. (2013) A survey of ex vivo/in vitro transduction efficiency of mammalian primary cells and cell lines with Nine natural adeno-associated virus (AAV1-9) and one engineered adeno-associated virus serotype. Virology Journal 10:1.
Crossref
Crossref
Arthur W. Nienhuis. (2013) Development of gene therapy for blood disorders: an update. Blood 122:9, pages 1556-1564.
Crossref
Crossref
Ketna Volcy & Nigel W. Fraser. (2013) DNA damage promotes herpes simplex virus-1 protein expression in a neuroblastoma cell line. Journal of NeuroVirology 19:1, pages 57-64.
Crossref
Crossref
Z. Nazir & S. Irshad. (2013) Stem cell – based gene therapy. Biopolymers and Cell 29:1, pages 21-32.
Crossref
Crossref
Shamim H. RahmanSylwia Bobis-WozowiczDebanjana ChatterjeeKatharina GellhausKaweh ParsRegine HeilbronnRoland JacobsToni Cathomen. (2013) The Nontoxic Cell Cycle Modulator Indirubin Augments Transduction of Adeno-Associated Viral Vectors and Zinc-Finger Nuclease-Mediated Gene Targeting. Human Gene Therapy 24:1, pages 67-77.
Crossref
Crossref
B L Ellis, M L Hirsch, S N Porter, R J Samulski & M H Porteus. (2012) Zinc-finger nuclease-mediated gene correction using single AAV vector transduction and enhancement by Food and Drug Administration-approved drugs. Gene Therapy 20:1, pages 35-42.
Crossref
Crossref
Brian R. Davis & Nicole L. Prokopishyn. 2013. Handbook of Stem Cells. Handbook of Stem Cells
937
949
.
Jean Charles Epinat, George H. Silva, Frédéric Pâques, Julianne Smith & Philippe Duchateau. 2013. Site-directed insertion of transgenes. Site-directed insertion of transgenes
147
185
.
Cherie L. Ramirez & J. Keith Joung. 2013. Site-directed insertion of transgenes. Site-directed insertion of transgenes
121
145
.
Yi Liu, Shangze Li, Huihui Zhang, Zurong Wan, Xiaodong Zhang & Runlei Du. (2012) A one-step cloning method for the construction of somatic cell gene targeting vectors: application to production of human knockout cell lines. BMC Biotechnology 12:1.
Crossref
Crossref
Nicole K. PaulkLaura Marquez LozaMilton J. FinegoldMarkus Grompe. (2012)
AAV-Mediated Gene Targeting Is Significantly Enhanced by Transient Inhibition of Nonhomologous End Joining or the Proteasome
In Vivo
. Human Gene Therapy 23:6, pages 658-665.
Crossref
Crossref
Yonglun Luo, Emil Kofod-Olsen, Rikke Christensen, Charlotte Brandt Sørensen & Lars Bolund. (2012) Targeted Genome Editing by Recombinant Adeno-Associated Virus (rAAV) Vectors for Generating Genetically Modified Pigs. Journal of Genetics and Genomics 39:6, pages 269-274.
Crossref
Crossref
Eva-Maria HändelKatharina GellhausKafaitullah KhanChristien BednarskiTatjana I. CornuFelix Müller-LerchRobert M. KotinRegine HeilbronnToni Cathomen. (2012) Versatile and Efficient Genome Editing in Human Cells by Combining Zinc-Finger Nucleases With Adeno-Associated Viral Vectors. Human Gene Therapy 23:3, pages 321-329.
Crossref
Crossref
Prashanth Asuri, Melissa A Bartel, Tandis Vazin, Jae-Hyung Jang, Tiffany B Wong & David V Schaffer. (2012) Directed Evolution of Adeno-associated Virus for Enhanced Gene Delivery and Gene Targeting in Human Pluripotent Stem Cells. Molecular Therapy 20:2, pages 329-338.
Crossref
Crossref
Daniel G. Miller. 2011. Adeno-Associated Virus. Adeno-Associated Virus
301
315
.
Béla Gyurcsik & Anikó Czene. (2011) Towards artificial metallonucleases for gene therapy: recent advances and new perspectives. Future Medicinal Chemistry 3:15, pages 1935-1966.
Crossref
Crossref
Araksya Izmiryan, Stéphane Basmaciogullari, Adrien Henry, Frédéric Paques & Olivier Danos. (2011) Efficient gene targeting mediated by a lentiviral vector-associated meganuclease. Nucleic Acids Research 39:17, pages 7610-7619.
Crossref
Crossref
Stefan Mockenhaupt & Dirk Grimm. (2011) Adeno-assoziierte Viren für effizientes Gene Targeting in humanen Zellen. BIOspektrum 17:5, pages 533-536.
Crossref
Crossref
Shamim H. RahmanMorgan L. MaederJ. Keith JoungToni Cathomen. (2011) Zinc-Finger Nucleases for Somatic Gene Therapy: The Next Frontier. Human Gene Therapy 22:8, pages 925-933.
Crossref
Crossref
Iram F Khan, Roli K Hirata & David W Russell. (2011) AAV-mediated gene targeting methods for human cells. Nature Protocols 6:4, pages 482-501.
Crossref
Crossref
Michael J. Metzger, Audrey McConnell-Smith, Barry L. Stoddard & A. Dusty Miller. (2011) Single-strand nicks induce homologous recombination with less toxicity than double-strand breaks using an AAV vector template. Nucleic Acids Research 39:3, pages 926-935.
Crossref
Crossref
M L Hirsch, L Green, M H Porteus & R J Samulski. (2010) Self-complementary AAV mediates gene targeting and enhances endonuclease delivery for double-strand break repair. Gene Therapy 17:9, pages 1175-1180.
Crossref
Crossref
Katharina GellhausTatjana I. CornuRegine HeilbronnToni Cathomen. (2010) Fate of Recombinant Adeno-Associated Viral Vector Genomes During DNA Double-Strand Break-Induced Gene Targeting in Human Cells. Human Gene Therapy 21:5, pages 543-553.
Crossref
Crossref
Lisa M Petek, David W Russell & Daniel G Miller. (2010) Frequent Endonuclease Cleavage at Off-target Locations In Vivo. Molecular Therapy 18:5, pages 983-986.
Crossref
Crossref
Sarah Radecke, Frank Radecke, Toni Cathomen & Klaus Schwarz. (2010) Zinc-finger Nuclease-induced Gene Repair With Oligodeoxynucleotides: Wanted and Unwanted Target Locus Modifications. Molecular Therapy 18:4, pages 743-753.
Crossref
Crossref
Aubrey D. N. J. de Grey. 2010. The Future of Aging. The Future of Aging
667
684
.
Shondra M. Pruett-Miller, David W. Reading, Shaina N. Porter & Matthew H. Porteus. (2009) Attenuation of Zinc Finger Nuclease Toxicity by Small-Molecule Regulation of Protein Levels. PLoS Genetics 5:2, pages e1000376.
Crossref
Crossref
Eva-Maria Händel, Stephen Alwin & Toni Cathomen. (2009) Expanding or Restricting the Target Site Repertoire of Zinc-finger Nucleases: The Inter-domain Linker as a Major Determinant of Target Site Selectivity. Molecular Therapy 17:1, pages 104-111.
Crossref
Crossref
Eli Berdougo, Marie-Emilie Terret & Prasad V. Jallepalli. 2009. Mitosis. Mitosis
21
37
.
J.-S. Kim, C. Bonifant, F. Bunz, W. S. Lane & T. Waldman. (2008) Epitope tagging of endogenous genes in diverse human cell lines. Nucleic Acids Research 36:19, pages e127-e127.
Crossref
Crossref
Toni Cathomen & J Keith Joung. (2008) Zinc-finger Nucleases: The Next Generation Emerges. Molecular Therapy 16:7, pages 1200-1207.
Crossref
Crossref
Brian R Schultz & Jeffrey S Chamberlain. (2008) Recombinant Adeno-associated Virus Transduction and Integration. Molecular Therapy 16:7, pages 1189-1199.
Crossref
Crossref
Christopher S. Rogers, Yanhong Hao, Tatiana Rokhlina, Melissa Samuel, David A. Stoltz, Yuhong Li, Elena Petroff, Daniel W. Vermeer, Amanda C. Kabel, Ziying Yan, Lee Spate, David Wax, Clifton N. Murphy, August Rieke, Kristin Whitworth, Michael L. Linville, Scott W. Korte, John F. Engelhardt, Michael J. Welsh & Randall S. Prather. (2008) Production of CFTR-null and CFTR-ΔF508 heterozygous pigs by adeno-associated virus–mediated gene targeting and somatic cell nuclear transfer. Journal of Clinical Investigation 118:4, pages 1571-1577.
Crossref
Crossref
Shondra M Pruett-Miller, Jon P Connelly, Morgan L Maeder, J Keith Joung & Matthew H Porteus. (2008) Comparison of Zinc Finger Nucleases for Use in Gene Targeting in Mammalian Cells. Molecular Therapy 16:4, pages 707-717.
Crossref
Crossref
Tatjana I Cornu, Stacey Thibodeau-Beganny, Eva Guhl, Stephen Alwin, Magdalena Eichtinger, JK Joung & Toni Cathomen. (2008) DNA-binding Specificity Is a Major Determinant of the Activity and Toxicity of Zinc-finger Nucleases. Molecular Therapy 16:2, pages 352-358.
Crossref
Crossref
Eric A. Hendrickson. 2008. Sourcebook of Models for Biomedical Research. Sourcebook of Models for Biomedical Research
509
525
.
Tatjana I Cornu & Toni Cathomen. (2007) Targeted Genome Modifications Using Integrase‐deficient Lentiviral Vectors. Molecular Therapy 15:12, pages 2107-2113.
Crossref
Crossref
Carlo Rago, Bert Vogelstein & Fred Bunz. (2007) Genetic knockouts and knockins in human somatic cells. Nature Protocols 2:11, pages 2734-2746.
Crossref
Crossref
Michal Szczepek, Vincent Brondani, Janine Büchel, Luis Serrano, David J Segal & Toni Cathomen. (2007) Structure-based redesign of the dimerization interface reduces the toxicity of zinc-finger nucleases. Nature Biotechnology 25:7, pages 786-793.
Crossref
Crossref
Marina Cavazzana-Calvo & Alain Fischer. (2007) Gene therapy for severe combined immunodeficiency: are we there yet?. Journal of Clinical Investigation 117:6, pages 1456-1465.
Crossref
Crossref
Toshihiko Hashizume & Noriaki Shimizu. (2007) Dissection of mammalian replicators by a novel plasmid stability assay. Journal of Cellular Biochemistry 101:3, pages 552-565.
Crossref
Crossref
ROGER BERTOLOTTI. (2007) EDITORIAL: "AUTOLOGOUS STEM CELL GENE THERAPY: TOWARD A UNIVERSAL PLATFORM FOR PERSONALIZED THERAPY". Gene Therapy and Regulation 03:01, pages 1-14.
Crossref
Crossref
Frank Radecke, Ingrid Peter, Sarah Radecke, Katharina Gellhaus, Klaus Schwarz & Toni Cathomen. (2006) Targeted Chromosomal Gene Modification in Human Cells by Single-Stranded Oligodeoxynucleotides in the Presence of a DNA Double-Strand Break. Molecular Therapy 14:6, pages 798-808.
Crossref
Crossref
Matthew H Porteus, Jon P Connelly & Shondra M Pruett. (2006) A Look to Future Directions in Gene Therapy Research for Monogenic Diseases. PLoS Genetics 2:9, pages e133.
Crossref
Crossref
Michael M. Seidman & John H. Wilson. 2006. Encyclopedia of Molecular Cell Biology and Molecular Medicine. Encyclopedia of Molecular Cell Biology and Molecular Medicine.
Melissa P. Knauert, Jennifer M. Kalish, Denise C. Hegan & Peter M. Glazer. (2006) Triplex-Stimulated Intermolecular Recombination at a Single-Copy Genomic Target. Molecular Therapy 14:3, pages 392-400.
Crossref
Crossref
John F Engelhardt. (2006) AAV hits the genomic bull's-eye. Nature Biotechnology 24:8, pages 949-950.
Crossref
Crossref
Vladan Ondřej, Stanislav Kozubek, Emílie Lukášová, Martin Falk, Pavel Matula, Petr Matula & Michal Kozubek. (2006) Directional motion of foreign plasmid DNA to nuclear HP1 foci. Chromosome Research 14:5, pages 505-514.
Crossref
Crossref
Matthew H. Porteus. (2006) Mammalian gene targeting with designed zinc finger nucleases. Molecular Therapy 13:2, pages 438-446.
Crossref
Crossref
Ana Vasileva & Rolf Jessberger. (2005) Precise hit: adeno-associated virus in gene targeting. Nature Reviews Microbiology 3:11, pages 837-847.
Crossref
Crossref
David A. Sorrell & Andreas F. Kolb. (2005) Targeted modification of mammalian genomes. Biotechnology Advances 23:7-8, pages 431-469.
Crossref
Crossref
Alfred S. Lewin, Peter M. Glazer & Leonard M. Milstone. (2005) Gene Therapy for Autosomal Dominant Disorders of Keratin. Journal of Investigative Dermatology Symposium Proceedings 10:1, pages 47-61.
Crossref
Crossref
T Cathomen & M D Weitzman. (2005) Gene Repair: Pointing the finger at genetic disease. Gene Therapy 12:19, pages 1415-1416.
Crossref
Crossref
Stephen Alwin, Maja B. Gere, Eva Guhl, Karin Effertz, Carlos F. BarbasIIIIII, David J. Segal, Matthew D. Weitzman & Toni Cathomen. (2005) Custom Zinc-Finger Nucleases for Use in Human Cells. Molecular Therapy 12:4, pages 610-617.
Crossref
Crossref
Craig J. Coates, Joseph M. Kaminski, James B. Summers, David J. Segal, Andrew D. Miller & Andreas F. Kolb. (2005) Site-directed genome modification: derivatives of DNA-modifying enzymes as targeting tools. Trends in Biotechnology 23:8, pages 407-419.
Crossref
Crossref
Paul C. Hendrie & David W. Russell. (2005) Gene Targeting with Viral Vectors. Molecular Therapy 12:1, pages 9-17.
Crossref
Crossref
Alain Fischer, Salima Hacein-bey-abina, Chantal Lagresle, Alexandrine Garrigue & Marina Cavazana-calvo. (2005) Thérapie génique des déficits immunitaires sévères : preuve de principe d’efficacité et problèmes soulevés. Thérapie génique, déficits immunitaires, rétrovirus, lentivirus, génome. Bulletin de l'Académie Nationale de Médecine 189:5, pages 779-788.
Crossref
Crossref
G. de Piédoue, R. Maurisse, I. Kuzniak, B. Lopez, A. Perrin, O. Nègre, P. Leboulch & J.-P. Feugeas. (2005) Improving gene replacement by intracellular formation of linear homologous DNA. The Journal of Gene Medicine 7:5, pages 649-656.
Crossref
Crossref
Grant TrobridgeRoli K. HirataDavid W. Russell. (2005) Gene Targeting by Adeno-Associated Virus Vectors Is Cell-Cycle Dependent. Human Gene Therapy 16:4, pages 522-526.
Crossref
Crossref
Douglas M. McCartySamuel M. YoungJr.Jr. & R. Jude Samulski. (2004) Integration of Adeno-Associated Virus (AAV) and Recombinant AAV Vectors. Annual Review of Genetics 38:1, pages 819-845.
Crossref
Crossref
Sung-Hae Lee Kang, Padraic P. Levings, Felicie Andersen, Philip J. Laipis, Kenneth I. Berns, Robert T. Zori & Jörg Bungert. (2004) Locus control region elements HS2 and HS3 in combination with chromatin boundaries confer high-level expression of a human β-globin transgene in a centromeric region. Genes to Cells 9:11, pages 1043-1053.
Crossref
Crossref
Alain Fischer, Salima Hacein-Bey-Abina & Marina Cavazzana-Calvo. (2004) Gene therapy for immunodeficiency diseases. Seminars in Hematology 41:4, pages 272-278.
Crossref
Crossref
Hiroaki Mon, Takahiro Kusakabe, Jae Man Lee, Yutaka Kawaguchi & Katsumi Koga. (2004) In vivo DNA double-strand breaks enhance gene targeting in cultured silkworm cells. Comparative Biochemistry and Physiology Part B: Biochemistry and Molecular Biology 139:1, pages 99-106.
Crossref
Crossref
Xiaoming Liu, Ziying Yan, Meihui Luo, Roman Zak, Ziyi Li, Ryan R. Driskell, Yumao Huang, Nam Tran & John F. Engelhardt. (2004) Targeted Correction of Single-Base-Pair Mutations with Adeno-Associated Virus Vectors under Nonselective Conditions. Journal of Virology 78:8, pages 4165-4175.
Crossref
Crossref
L.M. Pirzio, M.-A. Freulet-Marrière, Y. Bai, B. Fouladi, J.P. Murnane, L. Sabatier & C. Desmaze. (2004) Human fibroblasts expressing hTERT show remarkable karyotype stability even after exposure to ionizing radiation. Cytogenetic and Genome Research 104:1-4, pages 87-94.
Crossref
Crossref
Brian R. Davis & Nicole L. Prokopishyn. 2004. Handbook of Stem Cells. Handbook of Stem Cells
793
804
.
Francesca Storici, Christopher L. Durham, Dmitry A. Gordenin & Michael A. Resnick. (2003) Chromosomal site-specific double-strand breaks are efficiently targeted for repair by oligonucleotides in yeast. Proceedings of the National Academy of Sciences 100:25, pages 14994-14999.
Crossref
Crossref
Itaru Suzuki, Sungbin Im, Akihiro Tada, Greg Barsh, Vincent Hearing, Cathy Scott, Can Akcali, Mary Beth Davis & Zalfa Abdel-Malek. (1999) Participation of the Melanocortin-1 Receptor in the UV Control of Pigmentation. Journal of Investigative Dermatology Symposium Proceedings 4:1, pages 29-34.
Crossref
Crossref