Systemic Administration of Tripeptidyl Peptidase I in a Mouse Model of Late Infantile Neuronal Ceroid Lipofuscinosis: Effect of Glycan Modification
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May 31, 2026
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www.science.org/doi/10.1126/scitranslmed.adw9930?url_ver=Z39.88-2003&rfr_id=ori:rid:crossref.org&rfr_dat=cr_pub%20%200pubmed
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Enzyme replacement therapy for CLN1 batten disease that crosses the blood-brain-barrier
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Enhancing prenatal spinal surgery with stem cells
www.nature.com/articles/d41591-026-00017-3
Apr 9, 2026
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Blood-brain barrier disruption, traumatic encephalopathy, and cognitive decline in retired athletes
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Apr 9, 2026
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患者1人約3億円の遺伝子治療を「誰もが使える医療」へ、ノーベル賞科学者が挑むエコシステム構想 | Forbes JAPAN 公式サイト（フォーブス ジャパン）
forbesjapan.com/articles/detail/92463/page2
Mar 15, 2026
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YouTubeで効率的に学ぶ方法：科学に基づく動画学習ガイド | Glasp
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glasp.co/articles/how-to-take-smart-notes/ja
Mar 15, 2026
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Just a moment...
www.biorxiv.org/content/10.64898/2026.02.23.707565v1
Mar 9, 2026
An innovative hematopoietic stem cell gene therapy approach benefits CLN1 disease in the mouse model - EMBO Molecular Medicine
link.springer.com/article/10.15252/emmm.202215968
Feb 28, 2026
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Cause for Progressive Blindness Identified
www.genengnews.com/topics/translational-medicine/cause-for-progressive-blindness-identified/
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Oligodendrocyte Differentiation Holds Promise for MS Treatment Development
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Cell-type resolved protein atlas of brain lysosomes identifies SLC45A1-associated disease as a lysosomal disorder
www.cell.com/cell/fulltext/S0092-8674(25)01425-4?_returnURL=https%3A%2F%2Flinkinghub.elsevier.com%2Fretrieve%2Fpii%2FS0092867425014254%3Fshowall%3Dtrue
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Cell-Type Atlas of Brain Lysosomal Proteins May Clarify Neurological Disorders
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Jan 30, 2026
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Lysosomal Brain Protein Atlas
brainresilience.stanford.edu/lysosomal-brain-protein-atlas
Jan 30, 2026
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Treatment of Inherited Eye Defects by Systemic Hematopoietic Stem Cell Transplantation | IOVS | ARVO Journals
iovs.arvojournals.org/article.aspx?articleid=2469222
Jan 12, 2026
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Frontiers | The Contribution of Microglia to the Development and Maturation of the Visual System
www.frontiersin.org/journals/cellular-neuroscience/articles/10.3389/fncel.2021.659843/full
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www.researchgate.net/publication/336168617_Roles_of_Drug_Transporters_in_Blood-Retinal_Barrier/figures?lo=1&utm_medium=&utm_campaign=
Jan 12, 2026
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網膜 - 脳科学辞典
bsd.neuroinf.jp/wiki/%E7%B6%B2%E8%86%9C
Jan 12, 2026
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ミュラーグリア - 脳科学辞典
bsd.neuroinf.jp/wiki/%E3%83%9F%E3%83%A5%E3%83%A9%E3%83%BC%E3%82%B0%E3%83%AA%E3%82%A2
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視細胞 - 脳科学辞典
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網膜色素変性の過蛍光リング – 目医者情報
meisha.info/archives/744
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網膜色素変性 – 目医者情報
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CNS infection and immune privilege - Nature Reviews Neuroscience
www.nature.com/articles/s41583-018-0070-8
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2025 FDA approvals
www.nature.com/articles/d41573-026-00001-z
Jan 7, 2026
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BioMarin to buy Amicus Therapeutics for $4.8B, bringing together rare disease biotechs
www.statnews.com/2025/12/19/biomarin-amicus-acquisition-rare-disease-fabry-pompe/
Jan 5, 2026
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An AAV variant selected through NHP screens robustly transduces the brain and drives secreted protein expression in NHPs and mice
www.science.org/doi/10.1126/scitranslmed.adr2531
Jan 5, 2026
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Latus Bio Announces IND Clearance of LTS-101 for CLN2 Disease and Receipt of Fast Track, Orphan Drug, and Rare Pediatric Disease Designations
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Brain-Directed AAV Gene Therapy Rescues a Mouse Model of the CLN5 Form of Neuronal Ceroid Lipofuscinosis Disease and Normalizes a Blood Plasma Biomarker of Neurodegeneration | Human Gene Therapy
www.liebertpub.com/doi/10.1177/10430342251403448
Jan 4, 2026
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GEN Editors Reflect on Six of the Biggest Stories of the Year
www.genengnews.com/industry-news/gen-editors-reflect-on-six-of-the-biggest-stories-of-the-year/
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Purification and characterisation of a tripeptidyl aminopeptidase I from rat spleen
www.sciencedirect.com/science/article/abs/pii/S0167483898000120?via%3Dihub
Jan 2, 2026
1