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  • 1
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    Targeted genome editing in human repopulating haematopoietic stem cells (2014)
    Nature Publishing Group (NPG)
    Details
    Publication Date: 2014-05-30
    Description: Targeted genome editing by artificial nucleases has brought the goal of site-specific transgene integration and gene correction within the reach of gene therapy. However, its application to long-term repopulating haematopoietic stem cells (HSCs) has remained elusive. Here we show that poor permissiveness to gene transfer and limited proficiency of the homology-directed DNA repair pathway constrain gene targeting in human HSCs. By tailoring delivery platforms and culture conditions we overcame these barriers and provide stringent evidence of targeted integration in human HSCs by long-term multilineage repopulation of transplanted mice. We demonstrate the therapeutic potential of our strategy by targeting a corrective complementary DNA into the IL2RG gene of HSCs from healthy donors and a subject with X-linked severe combined immunodeficiency (SCID-X1). Gene-edited HSCs sustained normal haematopoiesis and gave rise to functional lymphoid cells that possess a selective growth advantage over those carrying disruptive IL2RG mutations. These results open up new avenues for treating SCID-X1 and other diseases.〈br /〉〈br /〉〈a href="https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4082311/" target="_blank"〉〈img src="https://static.pubmed.gov/portal/portal3rc.fcgi/4089621/img/3977009" border="0"〉〈/a〉   〈a href="https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4082311/" target="_blank"〉This paper as free author manuscript - peer-reviewed and accepted for publication〈/a〉〈br /〉〈br /〉〈span class="detail_caption"〉Notes: 〈/span〉Genovese, Pietro -- Schiroli, Giulia -- Escobar, Giulia -- Di Tomaso, Tiziano -- Firrito, Claudia -- Calabria, Andrea -- Moi, Davide -- Mazzieri, Roberta -- Bonini, Chiara -- Holmes, Michael C -- Gregory, Philip D -- van der Burg, Mirjam -- Gentner, Bernhard -- Montini, Eugenio -- Lombardo, Angelo -- Naldini, Luigi -- 249845/European Research Council/International -- TGT11D02/Telethon/Italy -- England -- Nature. 2014 Jun 12;510(7504):235-40. doi: 10.1038/nature13420. Epub 2014 May 28.〈br /〉〈span class="detail_caption"〉Author address: 〈/span〉TIGET, San Raffaele Telethon Institute for Gene Therapy, San Raffaele Scientific Institute, 20132 Milan, Italy. ; 1] TIGET, San Raffaele Telethon Institute for Gene Therapy, San Raffaele Scientific Institute, 20132 Milan, Italy [2] Vita Salute San Raffaele University, 20132 Milan, Italy. ; 1] TIGET, San Raffaele Telethon Institute for Gene Therapy, San Raffaele Scientific Institute, 20132 Milan, Italy [2] The University of Queensland Diamantina Institute, Translational Research Institute, Brisbane, Queensland 4102, Australia. ; Experimental Hematology Unit, San Raffaele Scientific Institute, 20132 Milan, Italy. ; Sangamo BioSciences Inc., Richmond, California 94804, USA. ; Department of Immunology Erasmus MC, University Medical Center, 3015 Rotterdam, The Netherlands. ; 1] TIGET, San Raffaele Telethon Institute for Gene Therapy, San Raffaele Scientific Institute, 20132 Milan, Italy [2] Vita Salute San Raffaele University, 20132 Milan, Italy [3].〈br /〉〈span class="detail_caption"〉Record origin:〈/span〉 〈a href="http://www.ncbi.nlm.nih.gov/pubmed/24870228" target="_blank"〉PubMed〈/a〉
    Keywords: Animals ; Antigens, CD34/metabolism ; DNA, Complementary/genetics ; Endonucleases/metabolism ; Fetal Blood/cytology/metabolism/transplantation ; Gene Targeting/*methods ; Genome, Human/*genetics ; Hematopoiesis/genetics ; Hematopoietic Stem Cell Transplantation ; Hematopoietic Stem Cells/*cytology/*metabolism ; Humans ; Interleukin Receptor Common gamma Subunit/genetics ; Male ; Mice ; Mutation/genetics ; Targeted Gene Repair/*methods ; X-Linked Combined Immunodeficiency Diseases/*genetics/therapy
    Print ISSN: 0028-0836
    Electronic ISSN: 1476-4687
    Topics: Biology , Chemistry and Pharmacology , Medicine , Natural Sciences in General , Physics
    Published by Nature Publishing Group (NPG)
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  • 2
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    Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome (2013)
    American Association for the Advancement of Science (AAAS)
    Details
    Publication Date: 2013-07-13
    Description: Wiskott-Aldrich syndrome (WAS) is an inherited immunodeficiency caused by mutations in the gene encoding WASP, a protein regulating the cytoskeleton. Hematopoietic stem/progenitor cell (HSPC) transplants can be curative, but, when matched donors are unavailable, infusion of autologous HSPCs modified ex vivo by gene therapy is an alternative approach. We used a lentiviral vector encoding functional WASP to genetically correct HSPCs from three WAS patients and reinfused the cells after a reduced-intensity conditioning regimen. All three patients showed stable engraftment of WASP-expressing cells and improvements in platelet counts, immune functions, and clinical scores. Vector integration analyses revealed highly polyclonal and multilineage haematopoiesis resulting from the gene-corrected HSPCs. Lentiviral gene therapy did not induce selection of integrations near oncogenes, and no aberrant clonal expansion was observed after 20 to 32 months. Although extended clinical observation is required to establish long-term safety, lentiviral gene therapy represents a promising treatment for WAS.〈br /〉〈br /〉〈a href="https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4375961/" target="_blank"〉〈img src="https://static.pubmed.gov/portal/portal3rc.fcgi/4089621/img/3977009" border="0"〉〈/a〉   〈a href="https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4375961/" target="_blank"〉This paper as free author manuscript - peer-reviewed and accepted for publication〈/a〉〈br /〉〈br /〉〈span class="detail_caption"〉Notes: 〈/span〉Aiuti, Alessandro -- Biasco, Luca -- Scaramuzza, Samantha -- Ferrua, Francesca -- Cicalese, Maria Pia -- Baricordi, Cristina -- Dionisio, Francesca -- Calabria, Andrea -- Giannelli, Stefania -- Castiello, Maria Carmina -- Bosticardo, Marita -- Evangelio, Costanza -- Assanelli, Andrea -- Casiraghi, Miriam -- Di Nunzio, Sara -- Callegaro, Luciano -- Benati, Claudia -- Rizzardi, Paolo -- Pellin, Danilo -- Di Serio, Clelia -- Schmidt, Manfred -- Von Kalle, Christof -- Gardner, Jason -- Mehta, Nalini -- Neduva, Victor -- Dow, David J -- Galy, Anne -- Miniero, Roberto -- Finocchi, Andrea -- Metin, Ayse -- Banerjee, Pinaki P -- Orange, Jordan S -- Galimberti, Stefania -- Valsecchi, Maria Grazia -- Biffi, Alessandra -- Montini, Eugenio -- Villa, Anna -- Ciceri, Fabio -- Roncarolo, Maria Grazia -- Naldini, Luigi -- R01 AI067946/AI/NIAID NIH HHS/ -- TGT11D01/Telethon/Italy -- TGT11D02/Telethon/Italy -- New York, N.Y. -- Science. 2013 Aug 23;341(6148):1233151. doi: 10.1126/science.1233151. Epub 2013 Jul 11.〈br /〉〈span class="detail_caption"〉Author address: 〈/span〉San Raffaele Telethon Institute for Gene Therapy, Division of Regenerative Medicine, Stem Cells, and Gene Therapy, San Raffaele Scientific Institute, 20132 Milan, Italy. aiuti.alessandro@hsr.it〈br /〉〈span class="detail_caption"〉Record origin:〈/span〉 〈a href="http://www.ncbi.nlm.nih.gov/pubmed/23845947" target="_blank"〉PubMed〈/a〉
    Keywords: Child ; Genetic Therapy/*methods ; Genetic Vectors ; *Hematopoietic Stem Cell Transplantation ; Hematopoietic Stem Cells/*metabolism ; Humans ; Lentivirus ; Male ; Transduction, Genetic ; Virus Integration ; Wiskott-Aldrich Syndrome/*therapy ; Wiskott-Aldrich Syndrome Protein/*genetics
    Print ISSN: 0036-8075
    Electronic ISSN: 1095-9203
    Topics: Biology , Chemistry and Pharmacology , Computer Science , Medicine , Natural Sciences in General , Physics
    Published by American Association for the Advancement of Science (AAAS)
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  • 3
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    Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy (2013)
    American Association for the Advancement of Science (AAAS)
    Details
    Publication Date: 2013-07-13
    Description: Metachromatic leukodystrophy (MLD) is an inherited lysosomal storage disease caused by arylsulfatase A (ARSA) deficiency. Patients with MLD exhibit progressive motor and cognitive impairment and die within a few years of symptom onset. We used a lentiviral vector to transfer a functional ARSA gene into hematopoietic stem cells (HSCs) from three presymptomatic patients who showed genetic, biochemical, and neurophysiological evidence of late infantile MLD. After reinfusion of the gene-corrected HSCs, the patients showed extensive and stable ARSA gene replacement, which led to high enzyme expression throughout hematopoietic lineages and in cerebrospinal fluid. Analyses of vector integrations revealed no evidence of aberrant clonal behavior. The disease did not manifest or progress in the three patients 7 to 21 months beyond the predicted age of symptom onset. These findings indicate that extensive genetic engineering of human hematopoiesis can be achieved with lentiviral vectors and that this approach may offer therapeutic benefit for MLD patients.〈br /〉〈span class="detail_caption"〉Notes: 〈/span〉Biffi, Alessandra -- Montini, Eugenio -- Lorioli, Laura -- Cesani, Martina -- Fumagalli, Francesca -- Plati, Tiziana -- Baldoli, Cristina -- Martino, Sabata -- Calabria, Andrea -- Canale, Sabrina -- Benedicenti, Fabrizio -- Vallanti, Giuliana -- Biasco, Luca -- Leo, Simone -- Kabbara, Nabil -- Zanetti, Gianluigi -- Rizzo, William B -- Mehta, Nalini A L -- Cicalese, Maria Pia -- Casiraghi, Miriam -- Boelens, Jaap J -- Del Carro, Ubaldo -- Dow, David J -- Schmidt, Manfred -- Assanelli, Andrea -- Neduva, Victor -- Di Serio, Clelia -- Stupka, Elia -- Gardner, Jason -- von Kalle, Christof -- Bordignon, Claudio -- Ciceri, Fabio -- Rovelli, Attilio -- Roncarolo, Maria Grazia -- Aiuti, Alessandro -- Sessa, Maria -- Naldini, Luigi -- TGT11B02/Telethon/Italy -- TGT11D02/Telethon/Italy -- New York, N.Y. -- Science. 2013 Aug 23;341(6148):1233158. doi: 10.1126/science.1233158. Epub 2013 Jul 11.〈br /〉〈span class="detail_caption"〉Author address: 〈/span〉San Raffaele Telethon Institute for Gene Therapy, San Raffaele Scientific Institute, 20132 Milan, Italy. biffi.alessandra@hsr.it〈br /〉〈span class="detail_caption"〉Record origin:〈/span〉 〈a href="http://www.ncbi.nlm.nih.gov/pubmed/23845948" target="_blank"〉PubMed〈/a〉
    Keywords: Brain/pathology ; Cerebroside-Sulfatase/*genetics ; DNA Damage ; Follow-Up Studies ; Genetic Engineering ; Genetic Therapy/*methods ; Genetic Vectors/toxicity ; *Hematopoietic Stem Cell Transplantation ; Hematopoietic Stem Cells/*metabolism ; Humans ; Lentivirus ; Leukodystrophy, Metachromatic/pathology/*therapy ; Magnetic Resonance Imaging ; Transduction, Genetic ; Treatment Outcome ; Virus Integration
    Print ISSN: 0036-8075
    Electronic ISSN: 1095-9203
    Topics: Biology , Chemistry and Pharmacology , Computer Science , Medicine , Natural Sciences in General , Physics
    Published by American Association for the Advancement of Science (AAAS)
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  • 4
    Electronic Resource
    Electronic Resource
    Structural and dynamical characterization of piroxicam by ^1H- and ^1^3C-NMR relaxation studies
    Amsterdam : Elsevier
    Biophysical Chemistry 27 (1987), S. 255-261 
    Details
    ISSN: 0301-4622
    Keywords: Internal motion ; Nuclear Overhauser effect ; Piroxicam ; ^1H-NMR ; ^1^3C-NMR
    Source: Elsevier Journal Backfiles on ScienceDirect 1907 - 2002
    Topics: Biology , Chemistry and Pharmacology , Physics
    Type of Medium: Electronic Resource
    URL: http://linkinghub.elsevier.com/retrieve/pii/0301-4622(87)80065-0
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    Paper (German National Licenses)
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