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Patient-specific midbrain organoids with CRISPR correction recapitulate neuronopathic Gaucher disease phenotypes and enable evaluation of novel therapies
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| Published in: | eLife |
|---|---|
| Format: | Online Article RSS Article |
| Published: |
2026
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| _version_ | 1868847855187263488 |
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| collection | WordPress RSS FRELIP Feed Integration |
| container_title | eLife |
| description | |
| discipline_display | Biology |
| discipline_facet | Biology |
| format | Online Article RSS Article |
| genre | Journal Article |
| id | rss_article:95291 |
| institution | FRELIP |
| journal_source_facet | eLife |
| last_indexed | 2026-06-24T03:36:40.561Z |
| publishDate | 2026 |
| publishDateSort | 2026 |
| record_format | rss_article |
| spellingShingle | Patient-specific midbrain organoids with CRISPR correction recapitulate neuronopathic Gaucher disease phenotypes and enable evaluation of novel therapies Biology General Biology |
| sub_discipline_display | General |
| sub_discipline_facet | General |
| subject_display | Biology General Biology |
| subject_facet | Biology General Biology |
| title | Patient-specific midbrain organoids with CRISPR correction recapitulate neuronopathic Gaucher disease phenotypes and enable evaluation of novel therapies |
| title_alt | Organoides de mesencéfalo específicos del paciente con corrección CRISPR recapitulan fenotipos de enfermedad de Gaucher neuronopática y permiten la evaluación de nuevas terapias Des organoïdes du mésencéphale spécifiques au patient avec correction CRISPR récapitulent les phénotypes de la maladie de Gaucher neuronopathique et permettent l'évaluation de nouvelles thérapies Organoides de mesencéfalo específicos do paciente com correção CRISPR recapitulam fenótipos da doença de Gaucher neuronopática e permitem avaliação de novas terapias |
| title_auth | Patient-specific midbrain organoids with CRISPR correction recapitulate neuronopathic Gaucher disease phenotypes and enable evaluation of novel therapies |
| title_es_txt | Organoides de mesencéfalo específicos del paciente con corrección CRISPR recapitulan fenotipos de enfermedad de Gaucher neuronopática y permiten la evaluación de nuevas terapias |
| title_fr_txt | Des organoïdes du mésencéphale spécifiques au patient avec correction CRISPR récapitulent les phénotypes de la maladie de Gaucher neuronopathique et permettent l'évaluation de nouvelles thérapies |
| title_full | Patient-specific midbrain organoids with CRISPR correction recapitulate neuronopathic Gaucher disease phenotypes and enable evaluation of novel therapies |
| title_fullStr | Patient-specific midbrain organoids with CRISPR correction recapitulate neuronopathic Gaucher disease phenotypes and enable evaluation of novel therapies |
| title_full_unstemmed | Patient-specific midbrain organoids with CRISPR correction recapitulate neuronopathic Gaucher disease phenotypes and enable evaluation of novel therapies |
| title_pt_txt | Organoides de mesencéfalo específicos do paciente com correção CRISPR recapitulam fenótipos da doença de Gaucher neuronopática e permitem avaliação de novas terapias |
| title_short | Patient-specific midbrain organoids with CRISPR correction recapitulate neuronopathic Gaucher disease phenotypes and enable evaluation of novel therapies |
| title_sort | patient-specific midbrain organoids with crispr correction recapitulate neuronopathic gaucher disease phenotypes and enable evaluation of novel therapies |
| topic | Biology General Biology |
| url | https://elifesciences.org/articles/109518 |