Selected publications:

Development of antisense oligonucleotide (ASO)-based strategies:

• Helm J, Schöls L, Hauser S. Towards Personalized Allele-Specific Antisense Oligonucleotide Therapies for Toxic Gain-of-Function Neurodegenerative Diseases. Pharmaceutics. 2022 Aug 16;14(8). doi: 10.3390/pharmaceutics14081708.
• Hauser S, Helm J, Kraft M, Korneck M, Hübener-Schmid J, Schöls L. Allele-specific targeting of mutant ataxin-3 by antisense oligonucleotides in SCA3-iPSC-derived neurons. Mol Ther Nucleic Acids. 2022 Mar 8;27:99-108.

Investigate preclinical treatment strategies for HSP and motor neuron disorders:

• Wiora L, Yuan Q, Hook S, Kraft M, Björkhem I, Ott M, Hauser S*, Schöls L*. AAV8 based gene replacement therapy for hereditary spastic paraplegia type 5. Mol Ther Methods Clin Dev. 2025
• Hauser S, Poenisch M, Schelling Y, Höflinger P, Schuster S, Teegler A, Betten R, Gustafsson JÅ, Hübener-Schmid J, Schlake T, Chevessier-Tünnesen F, Horscroft N, Björkhem I, Schöls L. mRNA as a Novel Treatment Strategy for Hereditary Spastic Paraplegia Type 5. Mol Ther Methods Clin Dev. 2019 Dec 13;15:359-370.
• Rehbach K, Kesavan J, Hauser S, Ritzenhofen S, Jungverdorben J, Schüle R, Schöls L, Peitz M, Brüstle O. Multiparametric rapid screening of neuronal process pathology for drug target identification in HSP patient-specific neurons. Sci Rep. 2019 Jul 3;9(1):9615.
• Schöls L, Rattay TW, Martus P, Meisner C, Baets J, Fischer I, Jägle C, Fraidakis MJ, Martinuzzi A, Saute JA, Scarlato M, Antenora A, Stendel C, Höflinger P, Lourenco CM, Abreu L, Smets K, Paucar M, Deconinck T, Bis DM, Wiethoff S, Bauer P, Arnoldi A, Marques W, Jardim LB, Hauser S, Criscuolo C, Filla A, Züchner S, Bassi MT, Klopstock T, De Jonghe P, Björkhem I, Schüle R. Hereditary spastic paraplegia type 5: natural history, biomarkers and a randomized controlled trial. Brain. 2017 Dec 1;140(12):3112-3127.

Development of iPSC-based disease models:

• Korneck M, Leonhardt A, Schöls L, Hauser S. Generation of homozygous and heterozygous REEP1 knockout induced pluripotent stem cell lines by CRISPR/Cas9 gene editing. Stem Cell Res. 2024 Mar 5;77:103378.
• Korneck M, Wiora L, Schöls L, Hauser S. Generation of two SPAST knockout human induced pluripotent stem cell lines to create a model for Hereditary Spastic Paraplegia type 4. Stem Cell Res. 2022 Apr;60:102741.
• Schuster S, Heuten E, Velic A, Admard J, Synofzik M, Ossowski S, Macek B, Hauser S, Schöls L. CHIP mutations affect the heat shock response differently in human fibroblasts and iPSC-derived neurons. Dis Model Mech. 2020 Oct 12;13(10).
• Hauser S, Schuster S, Heuten E, Höflinger P, Admard J, Schelling Y, Velic A, Macek B, Ossowski S, Schöls L. Comparative Transcriptional Profiling of Motor Neuron Disorder-Associated Genes in Various Human Cell Culture Models. Front Cell Dev Biol. 2020;8:544043.

Full list