de Boer H - Search Results

1

Small molecule 1a reduces FMRpolyG-mediated toxicity in in vitro and in vivo models for FMR1 premutation.

Haify SN, Buijsen RAM, Verwegen L, Severijnen LWFM, de Boer H, Boumeester V, Monshouwer R, Yang WY, Cameron MD, Willemsen R, Disney MD, Hukema RK. Haify SN, et al. Among authors: de boer h. Hum Mol Genet. 2021 Aug 12;30(17):1632-1648. doi: 10.1093/hmg/ddab143. Hum Mol Genet. 2021. PMID: 34077515 Free PMC article.

2

Combination Therapy in Fragile X Syndrome; Possibilities and Pitfalls Illustrated by Targeting the mGluR5 and GABA Pathway Simultaneously.

Zeidler S, de Boer H, Hukema RK, Willemsen R. Zeidler S, et al. Among authors: de boer h. Front Mol Neurosci. 2017 Nov 7;10:368. doi: 10.3389/fnmol.2017.00368. eCollection 2017. Front Mol Neurosci. 2017. PMID: 29163043 Free PMC article.

3

Paradoxical effect of baclofen on social behavior in the fragile X syndrome mouse model.

Zeidler S, Pop AS, Jaafar IA, de Boer H, Buijsen RAM, de Esch CEF, Nieuwenhuizen-Bakker I, Hukema RK, Willemsen R. Zeidler S, et al. Among authors: de boer h, de esch cef. Brain Behav. 2018 Jun;8(6):e00991. doi: 10.1002/brb3.991. Epub 2018 Apr 26. Brain Behav. 2018. PMID: 29785777 Free PMC article.

4

A missense variant in the nuclear export signal of the FMR1 gene causes intellectual disability.

Zeidler S, Severijnen LA, de Boer H, van der Toorn EC, Ruivenkamp CAL, Bijlsma EK, Willemsen R. Zeidler S, et al. Among authors: de boer h. Gene. 2021 Feb 5;768:145298. doi: 10.1016/j.gene.2020.145298. Epub 2020 Nov 9. Gene. 2021. PMID: 33181255

5

Pretransplant mobilization with granulocyte colony-stimulating factor improves B-cell reconstitution by lentiviral vector gene therapy in SCID-X1 mice.

Huston MW, Riegman AR, Yadak R, van Helsdingen Y, de Boer H, van Til NP, Wagemaker G. Huston MW, et al. Among authors: de boer h. Hum Gene Ther. 2014 Oct;25(10):905-14. doi: 10.1089/hum.2014.101. Hum Gene Ther. 2014. PMID: 25222508 Free PMC article.

6

Correction of murine Rag2 severe combined immunodeficiency by lentiviral gene therapy using a codon-optimized RAG2 therapeutic transgene.

van Til NP, de Boer H, Mashamba N, Wabik A, Huston M, Visser TP, Fontana E, Poliani PL, Cassani B, Zhang F, Thrasher AJ, Villa A, Wagemaker G. van Til NP, et al. Among authors: de boer h. Mol Ther. 2012 Oct;20(10):1968-80. doi: 10.1038/mt.2012.110. Epub 2012 Jun 12. Mol Ther. 2012. PMID: 22692499 Free PMC article.

7

Lentiviral Hematopoietic Stem Cell Gene Therapy Corrects Murine Pompe Disease.

Stok M, de Boer H, Huston MW, Jacobs EH, Roovers O, Visser TP, Jahr H, Duncker DJ, van Deel ED, Reuser AJJ, van Til NP, Wagemaker G. Stok M, et al. Among authors: de boer h. Mol Ther Methods Clin Dev. 2020 May 4;17:1014-1025. doi: 10.1016/j.omtm.2020.04.023. eCollection 2020 Jun 12. Mol Ther Methods Clin Dev. 2020. PMID: 32462050 Free PMC article.

8

A modified γ-retrovirus vector for X-linked severe combined immunodeficiency.

Hacein-Bey-Abina S, Pai SY, Gaspar HB, Armant M, Berry CC, Blanche S, Bleesing J, Blondeau J, de Boer H, Buckland KF, Caccavelli L, Cros G, De Oliveira S, Fernández KS, Guo D, Harris CE, Hopkins G, Lehmann LE, Lim A, London WB, van der Loo JC, Malani N, Male F, Malik P, Marinovic MA, McNicol AM, Moshous D, Neven B, Oleastro M, Picard C, Ritz J, Rivat C, Schambach A, Shaw KL, Sherman EA, Silberstein LE, Six E, Touzot F, Tsytsykova A, Xu-Bayford J, Baum C, Bushman FD, Fischer A, Kohn DB, Filipovich AH, Notarangelo LD, Cavazzana M, Williams DA, Thrasher AJ. Hacein-Bey-Abina S, et al. Among authors: de boer h, de oliveira s. N Engl J Med. 2014 Oct 9;371(15):1407-17. doi: 10.1056/NEJMoa1404588. N Engl J Med. 2014. PMID: 25295500 Free PMC article. Clinical Trial.

9

miRNA-embedded shRNAs for Lineage-specific BCL11A Knockdown and Hemoglobin F Induction.

Guda S, Brendel C, Renella R, Du P, Bauer DE, Canver MC, Grenier JK, Grimson AW, Kamran SC, Thornton J, de Boer H, Root DE, Milsom MD, Orkin SH, Gregory RI, Williams DA. Guda S, et al. Among authors: de boer h. Mol Ther. 2015 Sep;23(9):1465-74. doi: 10.1038/mt.2015.113. Epub 2015 Jun 17. Mol Ther. 2015. PMID: 26080908 Free PMC article.

10

Expanding the spectrum of recombination-activating gene 1 deficiency: a family with early-onset autoimmunity.

Henderson LA, Frugoni F, Hopkins G, de Boer H, Pai SY, Lee YN, Walter JE, Hazen MM, Notarangelo LD. Henderson LA, et al. Among authors: de boer h. J Allergy Clin Immunol. 2013 Oct;132(4):969-71.e1-2. doi: 10.1016/j.jaci.2013.06.032. Epub 2013 Jul 24. J Allergy Clin Immunol. 2013. PMID: 23891352 Free PMC article. No abstract available.

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