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Hematopoietic stem cell gene therapy for the treatment of SYNGAP1-related non-specific intellectual disability. J Gene Med. 2024 Jul; 26(7):e3717.
Anderson JS, Lodigiani AL, Barbaduomo CM, Beegle JR. PMID: 38967915.
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HumansAnimalsCells
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Functional rescue in an Angelman syndrome model following treatment with lentivector transduced hematopoietic stem cells. Hum Mol Genet. 2021 06 09; 30(12):1067-1083.
Adhikari A, Copping NA, Beegle J, Cameron DL, Deng P, O'Geen H, Segal DJ, Fink KD, Silverman JL, Anderson JS. PMID: 33856035; PMCID: PMC8188406.
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22 Fields:
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HumansAnimalsCells
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A novel Huntington's disease mouse model to assess the role of neuroinflammation on disease progression and to develop human cell therapies. Stem Cells Transl Med. 2021 07; 10(7):1033-1043.
Dahlenburg H, Cameron D, Yang S, Bachman A, Pollock K, Cary W, Pham M, Hendrix K, White J, Nelson H, Deng P, Anderson JS, Fink K, Nolta J. PMID: 33710799; PMCID: PMC8235129.
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4 Fields:
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HumansAnimals
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Improvement of motor and behavioral activity in Sandhoff mice transplanted with human CD34+ cells transduced with a HexA/HexB expressing lentiviral vector. J Gene Med. 2020 09; 22(9):e3205.
Beegle J, Hendrix K, Maciel H, Nolta JA, Anderson JS. PMID: 32335981.
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8 Fields:
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HumansAnimalsCells
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Allele-Specific Reduction of the Mutant Huntingtin Allele Using Transcription Activator-Like Effectors in Human Huntington's Disease Fibroblasts. Cell Transplant. 2016; 25(4):677-86.
Fink KD, Deng P, Gutierrez J, Anderson JS, Torrest A, Komarla A, Kalomoiris S, Cary W, Anderson JD, Gruenloh W, Duffy A, Tempkin T, Annett G, Wheelock V, Segal DJ, Nolta JA. PMID: 26850319; PMCID: PMC6476541.
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27 Fields:
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HumansCells
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Safety and efficacy of a tCD25 preselective combination anti-HIV lentiviral vector in human hematopoietic stem and progenitor cells. Stem Cells. 2015 Mar; 33(3):870-9.
Barclay SL, Yang Y, Zhang S, Fong R, Barraza A, Nolta JA, Torbett BE, Abedi M, Bauer G, Anderson JS. PMID: 25524029.
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9 Fields:
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HumansAnimalsCells
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Canine epidermal neural crest stem cells: characterization and potential as therapy candidate for a large animal model of spinal cord injury. Stem Cells Transl Med. 2014 Mar; 3(3):334-45.
Gericota B, Anderson JS, Mitchell G, Borjesson DL, Sturges BK, Nolta JA, Sieber-Blum M. PMID: 24443004; PMCID: PMC3952930.
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8 Fields:
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HumansAnimalsCells
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Stem cell transplantation in the context of HIV--how can we cure HIV infection? Expert Rev Clin Immunol. 2014 Jan; 10(1):107-16.
Bauer G, Anderson JS. PMID: 24308835.
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Using TRIM5α as an HIV therapeutic: the alpha gene? Expert Opin Biol Ther. 2013 Jul; 13(7):1029-38.
Anderson JS. PMID: 23480791.
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2 Fields:
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HumansAnimalsCells
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CD25 preselective anti-HIV vectors for improved HIV gene therapy. Hum Gene Ther Methods. 2012 Dec; 23(6):366-75.
Kalomoiris S, Lawson J, Chen RX, Bauer G, Nolta JA, Anderson JS. PMID: 23216020; PMCID: PMC4015076.
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3 Fields:
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Fighting HIV with stem cell therapy: one step closer to human trials? Expert Rev Anti Infect Ther. 2012 Oct; 10(10):1071-3.
Anderson JS, Bauer G. PMID: 23199392.
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Humans
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Generation of an HIV-1-resistant immune system with CD34(+) hematopoietic stem cells transduced with a triple-combination anti-HIV lentiviral vector. J Virol. 2012 May; 86(10):5719-29.
Walker JE, Chen RX, McGee J, Nacey C, Pollard RB, Abedi M, Bauer G, Nolta JA, Anderson JS. PMID: 22398281; PMCID: PMC3347262.
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45 Fields:
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HumansAnimalsCells
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Generation of HIV-1 resistant and functional macrophages from hematopoietic stem cell-derived induced pluripotent stem cells. Mol Ther. 2011 Mar; 19(3):584-93.
Kambal A, Mitchell G, Cary W, Gruenloh W, Jung Y, Kalomoiris S, Nacey C, McGee J, Lindsey M, Fury B, Bauer G, Nolta JA, Anderson JS. PMID: 21119622; PMCID: PMC3048185.
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36 Fields:
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HumansCells
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Specific transduction of HIV-susceptible cells for CCR5 knockdown and resistance to HIV infection: a novel method for targeted gene therapy and intracellular immunization. J Acquir Immune Defic Syndr. 2009 Oct 01; 52(2):152-61.
Anderson JS, Walker J, Nolta JA, Bauer G. PMID: 19593160; PMCID: PMC3777721.
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13 Fields:
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HumansAnimalsCells
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Preintegration HIV-1 inhibition by a combination lentiviral vector containing a chimeric TRIM5 alpha protein, a CCR5 shRNA, and a TAR decoy. Mol Ther. 2009 Dec; 17(12):2103-14.
Anderson JS, Javien J, Nolta JA, Bauer G. PMID: 19690520; PMCID: PMC2814390.
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33 Fields:
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HumansAnimalsCells
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Human immunodeficiency virus type 1 restriction by human-rhesus chimeric tripartite motif 5alpha (TRIM 5alpha) in CD34(+) cell-derived macrophages in vitro and in T cells in vivo in severe combined immunodeficient (SCID-hu) mice transplanted with human fetal tissue. Hum Gene Ther. 2008 Mar; 19(3):217-28.
Anderson J, Akkina R. PMID: 18279037.
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27 Fields:
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HumansAnimalsCells
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Complete knockdown of CCR5 by lentiviral vector-expressed siRNAs and protection of transgenic macrophages against HIV-1 infection. Gene Ther. 2007 Sep; 14(17):1287-97.
Anderson J, Akkina R. PMID: 17597795.
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27 Fields:
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HumansCells
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Safety and efficacy of a lentiviral vector containing three anti-HIV genes--CCR5 ribozyme, tat-rev siRNA, and TAR decoy--in SCID-hu mouse-derived T cells. Mol Ther. 2007 Jun; 15(6):1182-8.
Anderson J, Li MJ, Palmer B, Remling L, Li S, Yam P, Yee JK, Rossi J, Zaia J, Akkina R. PMID: 17406343.
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55 Fields:
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HumansAnimalsCells
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Short-hairpin RNA expressed from polymerase III promoters mediates RNA interference in mosquito cells. Insect Mol Biol. 2007 Apr; 16(2):199-206.
Konet DS, Anderson J, Piper J, Akkina R, Suchman E, Carlson J. PMID: 17298556.
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19 Fields:
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AnimalsCells
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Roles of human parainfluenza virus type 3 bases 13 to 78 in replication and transcription: identification of an additional replication promoter element and evidence for internal transcription initiation. J Virol. 2006 Jun; 80(11):5388-96.
Hoffman MA, Thorson LM, Vickman JE, Anderson JS, May NA, Schweitzer MN. PMID: 16699019; PMCID: PMC1472146.
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PubMed Mentions:
2 Fields:
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HumansCells
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Ex vivo selection and expansion of cells based on expression of a mutated inosine monophosphate dehydrogenase 2 after HIV vector transduction: effects on lymphocytes, monocytes, and CD34+ stem cells. Mol Ther. 2006 Aug; 14(2):236-44.
Yam P, Jensen M, Akkina R, Anderson J, Villacres MC, Wu J, Zaia JA, Yee JK. PMID: 16647299.
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12 Fields:
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HumansAnimalsCells
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Derivation of normal macrophages from human embryonic stem (hES) cells for applications in HIV gene therapy. Retrovirology. 2006 Apr 19; 3:24.
Anderson JS, Bandi S, Kaufman DS, Akkina R. PMID: 16623949; PMCID: PMC1462997.
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27 Fields:
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HumansCells
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TRIM5alpharh expression restricts HIV-1 infection in lentiviral vector-transduced CD34+-cell-derived macrophages. Mol Ther. 2005 Oct; 12(4):687-96.
Anderson J, Akkina R. PMID: 16081321.
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14 Fields:
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HumansCells
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Long-term inhibition of HIV-1 infection in primary hematopoietic cells by lentiviral vector delivery of a triple combination of anti-HIV shRNA, anti-CCR5 ribozyme, and a nucleolar-localizing TAR decoy. Mol Ther. 2005 Nov; 12(5):900-9.
Li MJ, Kim J, Li S, Zaia J, Yee JK, Anderson J, Akkina R, Rossi JJ. PMID: 16115802.
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PubMed Mentions:
107 Fields:
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HumansCells
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CXCR4 and CCR5 shRNA transgenic CD34+ cell derived macrophages are functionally normal and resist HIV-1 infection. Retrovirology. 2005 Aug 18; 2:53.
Anderson J, Akkina R. PMID: 16109172; PMCID: PMC1199620.
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25 Fields:
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HumansCells
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Potent suppression of HIV type 1 infection by a short hairpin anti-CXCR4 siRNA. AIDS Res Hum Retroviruses. 2003 Aug; 19(8):699-706.
Anderson J, Banerjea A, Planelles V, Akkina R. PMID: 13678472.
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27 Fields:
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HumansCells
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siRNAs, ribozymes and RNA decoys in modeling stem cell-based gene therapy for HIV/AIDS. Anticancer Res. 2003 May-Jun; 23(3A):1997-2005.
Akkina R, Banerjea A, Bai J, Anderson J, Li MJ, Rossi J. PMID: 12894572.
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17 Fields:
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HumansAnimalsCells
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Bispecific short hairpin siRNA constructs targeted to CD4, CXCR4, and CCR5 confer HIV-1 resistance. Oligonucleotides. 2003; 13(5):303-12.
Anderson J, Banerjea A, Akkina R. PMID: 15000821.
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PubMed Mentions:
41 Fields:
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HumansAnimalsCells