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  1. Book ; Thesis: Characterization of conformational changes in the catalytic core of Gamma-secretase

    Wahlster, Lara

    implications for Alzheimer's disease pathology and normal aging

    2012  

    Author's details vorgelegt von Lara Wahlster
    Language English
    Size X, 132 S. : Ill., graph. Darst.
    Publishing country Germany
    Document type Book ; Thesis
    Thesis / German Habilitation thesis Heidelberg, Univ., Diss., 2013
    HBZ-ID HT017823026
    Database Catalogue ZB MED Medicine, Health

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    In stock of ZB MED Cologne/Königswinter

    Shelf markLoan statusLocation
    2013 E 1005 order for loan Magazin

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  2. Article: Editorial: Advances in predisposition to bone marrow failure and hematopoietic neoplasms.

    Sahoo, Sushree S / Abdelhamed, Sherif / Mochizuki-Kashio, Makiko / Wahlster, Lara

    Frontiers in oncology

    2024  Volume 14, Page(s) 1377974

    Language English
    Publishing date 2024-02-16
    Publishing country Switzerland
    Document type Editorial
    ZDB-ID 2649216-7
    ISSN 2234-943X
    ISSN 2234-943X
    DOI 10.3389/fonc.2024.1377974
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  3. Article ; Online: I SPI1 something needed for B cells.

    Wahlster, Lara / Sankaran, Vijay G

    The Journal of experimental medicine

    2021  Volume 218, Issue 7

    Abstract: In this issue, Le Coz et al. (2021. J. Exp. Med.https://doi.org/10.1084/jem.20201750) describe a novel immunodeficiency syndrome caused by mutations in SPI1. Through a series of in-depth studies, the authors provide insights into how SPI1 affects blood ... ...

    Abstract In this issue, Le Coz et al. (2021. J. Exp. Med.https://doi.org/10.1084/jem.20201750) describe a novel immunodeficiency syndrome caused by mutations in SPI1. Through a series of in-depth studies, the authors provide insights into how SPI1 affects blood lineage specification, highlighting the important role of master transcription factors as cellular fate determinants.
    MeSH term(s) B-Lymphocytes
    Language English
    Publishing date 2021-05-12
    Publishing country United States
    Document type Journal Article ; Comment
    ZDB-ID 218343-2
    ISSN 1540-9538 ; 0022-1007
    ISSN (online) 1540-9538
    ISSN 0022-1007
    DOI 10.1084/jem.20210572
    Database MEDical Literature Analysis and Retrieval System OnLINE

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    Ua VI Zs.107: Show issues Location:
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    ab Jg. 2022: Lesesaal (EG)
    Zs.MG 45: Show issues

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  4. Article ; Online: Progress towards generation of human haematopoietic stem cells.

    Wahlster, Lara / Daley, George Q

    Nature cell biology

    2016  Volume 18, Issue 11, Page(s) 1111–1117

    Abstract: De novo generation of haematopoietic stem cells from different human pluripotent stem cell sources remains a high priority for haematology and regenerative medicine. At present, efficient derivation of functional haematopoietic stem cells with the ... ...

    Abstract De novo generation of haematopoietic stem cells from different human pluripotent stem cell sources remains a high priority for haematology and regenerative medicine. At present, efficient derivation of functional haematopoietic stem cells with the capability for definitive in vivo engraftment and multi-lineage potential remains challenging. Here, we discuss recent progress and strategies to overcome obstacles that have thwarted past efforts. In addition, we review promising advances in the generation of mature blood lineages and the potential of induced pluripotent stem cells.
    MeSH term(s) Cell Differentiation/physiology ; Cell- and Tissue-Based Therapy ; Embryonic Stem Cells/cytology ; Hematopoietic Stem Cells/cytology ; Humans ; Pluripotent Stem Cells/cytology ; Regenerative Medicine
    Language English
    Publishing date 2016-11
    Publishing country England
    Document type Journal Article ; Review
    ZDB-ID 1474722-4
    ISSN 1476-4679 ; 1465-7392
    ISSN (online) 1476-4679
    ISSN 1465-7392
    DOI 10.1038/ncb3419
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    Zs.A 5169: Show issues Location:
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    Jg. 1995 - 2021: Lesesall (2.OG)
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    Zs.MG 12: Show issues

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  5. Article ; Online: Modeling Parkinson's disease in a dish--a story of yeast and men.

    Ebrahimi-Fakhari, Darius / Wahlster, Lara

    Movement disorders : official journal of the Movement Disorder Society

    2014  Volume 29, Issue 1, Page(s) 34

    MeSH term(s) Animals ; Benzimidazoles/pharmacology ; Female ; Humans ; Neurons/drug effects ; Parkinson Disease/metabolism ; alpha-Synuclein/metabolism
    Chemical Substances Benzimidazoles ; alpha-Synuclein
    Language English
    Publishing date 2014-01
    Publishing country United States
    Document type Comment ; Journal Article
    ZDB-ID 607633-6
    ISSN 1531-8257 ; 0885-3185
    ISSN (online) 1531-8257
    ISSN 0885-3185
    DOI 10.1002/mds.25781
    Database MEDical Literature Analysis and Retrieval System OnLINE

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    Zs.A 2555: Show issues Location:
    Je nach Verfügbarkeit (siehe Angabe bei Bestand)
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    Jg. 1995 - 2021: Lesesall (2.OG)
    ab Jg. 2022: Lesesaal (EG)
    Zs.MO 238: Show issues

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  6. Article ; Online: COVID-19 presenting with autoimmune hemolytic anemia in the setting of underlying immune dysregulation.

    Wahlster, Lara / Weichert-Leahey, Nina / Trissal, Maria / Grace, Rachael F / Sankaran, Vijay G

    Pediatric blood & cancer

    2020  Volume 67, Issue 9, Page(s) e28382

    MeSH term(s) Adolescent ; Adrenal Cortex Hormones/administration & dosage ; Anemia, Hemolytic, Autoimmune/blood ; Anemia, Hemolytic, Autoimmune/diagnosis ; Anemia, Hemolytic, Autoimmune/pathology ; Anemia, Hemolytic, Autoimmune/therapy ; Benzoates/administration & dosage ; Betacoronavirus/metabolism ; COVID-19 ; Coronavirus Infections/blood ; Coronavirus Infections/diagnosis ; Coronavirus Infections/pathology ; Coronavirus Infections/therapy ; Erythrocyte Transfusion ; Humans ; Hydrazines/administration & dosage ; Male ; Mycophenolic Acid/administration & dosage ; Oxygen/administration & dosage ; Pandemics ; Pneumonia, Viral/blood ; Pneumonia, Viral/diagnosis ; Pneumonia, Viral/pathology ; Pneumonia, Viral/therapy ; Pyrazoles/administration & dosage ; SARS-CoV-2
    Chemical Substances Adrenal Cortex Hormones ; Benzoates ; Hydrazines ; Pyrazoles ; Mycophenolic Acid (HU9DX48N0T) ; eltrombopag (S56D65XJ9G) ; Oxygen (S88TT14065)
    Keywords covid19
    Language English
    Publishing date 2020-06-03
    Publishing country United States
    Document type Case Reports ; Letter ; Research Support, N.I.H., Extramural
    ZDB-ID 2131448-2
    ISSN 1545-5017 ; 1545-5009
    ISSN (online) 1545-5017
    ISSN 1545-5009
    DOI 10.1002/pbc.28382
    Database MEDical Literature Analysis and Retrieval System OnLINE

    Full text online

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    Zs.A 1131: Show issues Location:
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  7. Article ; Online: High-content screening identifies a small molecule that restores AP-4-dependent protein trafficking in neuronal models of AP-4-associated hereditary spastic paraplegia.

    Saffari, Afshin / Brechmann, Barbara / Böger, Cedric / Saber, Wardiya Afshar / Jumo, Hellen / Whye, Dosh / Wood, Delaney / Wahlster, Lara / Alecu, Julian E / Ziegler, Marvin / Scheffold, Marlene / Winden, Kellen / Hubbs, Jed / Buttermore, Elizabeth D / Barrett, Lee / Borner, Georg H H / Davies, Alexandra K / Ebrahimi-Fakhari, Darius / Sahin, Mustafa

    Nature communications

    2024  Volume 15, Issue 1, Page(s) 584

    Abstract: Unbiased phenotypic screens in patient-relevant disease models offer the potential to detect therapeutic targets for rare diseases. In this study, we developed a high-throughput screening assay to identify molecules that correct aberrant protein ... ...

    Abstract Unbiased phenotypic screens in patient-relevant disease models offer the potential to detect therapeutic targets for rare diseases. In this study, we developed a high-throughput screening assay to identify molecules that correct aberrant protein trafficking in adapter protein complex 4 (AP-4) deficiency, a rare but prototypical form of childhood-onset hereditary spastic paraplegia characterized by mislocalization of the autophagy protein ATG9A. Using high-content microscopy and an automated image analysis pipeline, we screened a diversity library of 28,864 small molecules and identified a lead compound, BCH-HSP-C01, that restored ATG9A pathology in multiple disease models, including patient-derived fibroblasts and induced pluripotent stem cell-derived neurons. We used multiparametric orthogonal strategies and integrated transcriptomic and proteomic approaches to delineate potential mechanisms of action of BCH-HSP-C01. Our results define molecular regulators of intracellular ATG9A trafficking and characterize a lead compound for the treatment of AP-4 deficiency, providing important proof-of-concept data for future studies.
    MeSH term(s) Humans ; Spastic Paraplegia, Hereditary/drug therapy ; Spastic Paraplegia, Hereditary/genetics ; Spastic Paraplegia, Hereditary/metabolism ; Proteomics ; Neurons/metabolism ; Protein Transport ; Proteins/metabolism ; Mutation
    Chemical Substances Proteins
    Language English
    Publishing date 2024-01-17
    Publishing country England
    Document type Journal Article
    ZDB-ID 2553671-0
    ISSN 2041-1723 ; 2041-1723
    ISSN (online) 2041-1723
    ISSN 2041-1723
    DOI 10.1038/s41467-023-44264-1
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  8. Article ; Online: A noncoding regulatory variant in IKZF1 increases acute lymphoblastic leukemia risk in Hispanic/Latino children.

    de Smith, Adam J / Wahlster, Lara / Jeon, Soyoung / Kachuri, Linda / Black, Susan / Langie, Jalen / Cato, Liam D / Nakatsuka, Nathan / Chan, Tsz-Fung / Xia, Guangze / Mazumder, Soumyaa / Yang, Wenjian / Gazal, Steven / Eng, Celeste / Hu, Donglei / Burchard, Esteban González / Ziv, Elad / Metayer, Catherine / Mancuso, Nicholas /
    Yang, Jun J / Ma, Xiaomei / Wiemels, Joseph L / Yu, Fulong / Chiang, Charleston W K / Sankaran, Vijay G

    Cell genomics

    2024  Volume 4, Issue 4, Page(s) 100526

    Abstract: Hispanic/Latino children have the highest risk of acute lymphoblastic leukemia (ALL) in the US compared to other racial/ethnic groups, yet the basis of this remains incompletely understood. Through genetic fine-mapping analyses, we identified a new ... ...

    Abstract Hispanic/Latino children have the highest risk of acute lymphoblastic leukemia (ALL) in the US compared to other racial/ethnic groups, yet the basis of this remains incompletely understood. Through genetic fine-mapping analyses, we identified a new independent childhood ALL risk signal near IKZF1 in self-reported Hispanic/Latino individuals, but not in non-Hispanic White individuals, with an effect size of ∼1.44 (95% confidence interval = 1.33-1.55) and a risk allele frequency of ∼18% in Hispanic/Latino populations and <0.5% in European populations. This risk allele was positively associated with Indigenous American ancestry, showed evidence of selection in human history, and was associated with reduced IKZF1 expression. We identified a putative causal variant in a downstream enhancer that is most active in pro-B cells and interacts with the IKZF1 promoter. This variant disrupts IKZF1 autoregulation at this enhancer and results in reduced enhancer activity in B cell progenitors. Our study reveals a genetic basis for the increased ALL risk in Hispanic/Latino children.
    MeSH term(s) Humans ; Child ; Genetic Predisposition to Disease/genetics ; Polymorphism, Single Nucleotide ; Transcription Factors/genetics ; Precursor Cell Lymphoblastic Leukemia-Lymphoma/genetics ; Hispanic or Latino/genetics ; Ikaros Transcription Factor/genetics
    Chemical Substances Transcription Factors ; IKZF1 protein, human ; Ikaros Transcription Factor (148971-36-2)
    Language English
    Publishing date 2024-03-26
    Publishing country United States
    Document type Journal Article
    ISSN 2666-979X
    ISSN (online) 2666-979X
    DOI 10.1016/j.xgen.2024.100526
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  9. Article: High-Content Small Molecule Screen Identifies a Novel Compound That Restores AP-4-Dependent Protein Trafficking in Neuronal Models of AP-4-Associated Hereditary Spastic Paraplegia.

    Saffari, Afshin / Brechmann, Barbara / Boeger, Cedric / Saber, Wardiya Afshar / Jumo, Hellen / Whye, Dosh / Wood, Delaney / Wahlster, Lara / Alecu, Julian / Ziegler, Marvin / Scheffold, Marlene / Winden, Kellen / Hubbs, Jed / Buttermore, Elizabeth / Barrett, Lee / Borner, Georg / Davies, Alexandra / Sahin, Mustafa / Ebrahimi-Fakhari, Darius

    Research square

    2023  

    Abstract: Unbiased phenotypic screens in patient-relevant disease models offer the potential to detect novel therapeutic targets for rare diseases. In this study, we developed a high-throughput screening assay to identify molecules that correct aberrant protein ... ...

    Abstract Unbiased phenotypic screens in patient-relevant disease models offer the potential to detect novel therapeutic targets for rare diseases. In this study, we developed a high-throughput screening assay to identify molecules that correct aberrant protein trafficking in adaptor protein complex 4 (AP-4) deficiency, a rare but prototypical form of childhood-onset hereditary spastic paraplegia, characterized by mislocalization of the autophagy protein ATG9A. Using high-content microscopy and an automated image analysis pipeline, we screened a diversity library of 28,864 small molecules and identified a lead compound,
    Language English
    Publishing date 2023-06-12
    Publishing country United States
    Document type Preprint
    DOI 10.21203/rs.3.rs-3036166/v1
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  10. Article ; Online: Restoring impaired protein metabolism in Parkinson's disease--TFEB-mediated autophagy as a novel therapeutic target.

    Ebrahimi-Fakhari, Darius / Wahlster, Lara

    Movement disorders : official journal of the Movement Disorder Society

    2013  Volume 28, Issue 10, Page(s) 1346

    MeSH term(s) Animals ; Autophagy ; Basic Helix-Loop-Helix Leucine Zipper Transcription Factors/physiology ; Female ; Humans ; Mesencephalon/pathology ; Neurons/metabolism ; alpha-Synuclein/metabolism
    Chemical Substances Basic Helix-Loop-Helix Leucine Zipper Transcription Factors ; alpha-Synuclein
    Language English
    Publishing date 2013-09
    Publishing country United States
    Document type Comment ; Journal Article
    ZDB-ID 607633-6
    ISSN 1531-8257 ; 0885-3185
    ISSN (online) 1531-8257
    ISSN 0885-3185
    DOI 10.1002/mds.25601
    Database MEDical Literature Analysis and Retrieval System OnLINE

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    Zs.MO 238: Show issues

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