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/
Feb 21, 2026
1
Oligodendrocyte Differentiation Holds Promise for MS Treatment Development
www.genengnews.com/topics/translational-medicine/oligodendrocyte-differentiation-holds-promise-for-ms-treatment-development/
Jan 30, 2026
11
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
Jan 30, 2026
1
Cell-Type Atlas of Brain Lysosomal Proteins May Clarify Neurological Disorders
www.genengnews.com/topics/translational-medicine/cell-type-atlas-of-brain-lysosomal-proteins-may-clarify-neurological-disorders/
Jan 30, 2026
41
Lysosomal Brain Protein Atlas
brainresilience.stanford.edu/lysosomal-brain-protein-atlas
Jan 30, 2026
1
Treatment of Inherited Eye Defects by Systemic Hematopoietic Stem Cell Transplantation | IOVS | ARVO Journals
iovs.arvojournals.org/article.aspx?articleid=2469222
Jan 12, 2026
21
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
Jan 12, 2026
99
(PDF) Roles of Drug Transporters in Blood-Retinal Barrier
www.researchgate.net/publication/336168617_Roles_of_Drug_Transporters_in_Blood-Retinal_Barrier/figures?lo=1&utm_medium=&utm_campaign=
Jan 12, 2026
1
網膜 - 脳科学辞典
bsd.neuroinf.jp/wiki/%E7%B6%B2%E8%86%9C
Jan 12, 2026
3
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
Jan 12, 2026
21
視細胞 - 脳科学辞典
bsd.neuroinf.jp/wiki/%E8%A6%96%E7%B4%B0%E8%83%9E
Jan 12, 2026
21
網膜色素変性の過蛍光リング – 目医者情報
meisha.info/archives/744
Jan 12, 2026
1
網膜色素変性 – 目医者情報
meisha.info/archives/726
Jan 12, 2026
11
CNS infection and immune privilege - Nature Reviews Neuroscience
www.nature.com/articles/s41583-018-0070-8
Jan 12, 2026
11
2025 FDA approvals
www.nature.com/articles/d41573-026-00001-z
Jan 7, 2026
1
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
1
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
22
Latus Bio Announces IND Clearance of LTS-101 for CLN2 Disease and Receipt of Fast Track, Orphan Drug, and Rare Pediatric Disease Designations
www.businesswire.com/news/home/20251202268130/en/Latus-Bio-Announces-IND-Clearance-of-LTS-101-for-CLN2-Disease-and-Receipt-of-Fast-Track-Orphan-Drug-and-Rare-Pediatric-Disease-Designations
Jan 5, 2026
53
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
21
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/
Jan 2, 2026
2220
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
Structure of Tripeptidyl-peptidase I Provides Insight into the Molecular Basis of Late Infantile Neuronal Ceroid Lipofuscinosis *
www.jbc.org/article/S0021-9258(19)81812-4/fulltext
Jan 2, 2026
41
Staying Current in Data Science and Computational Biology: 2026 Edition
blog.stephenturner.us/p/staying-current-in-data-science-and-computational-biology-2026
Jan 2, 2026
Inciteful+Zotero to find relevant literature
blog.stephenturner.us/p/inciteful-zotero-biologpt-semantic-scholar
Jan 2, 2026
11
資料112－1 ミトコンドリア置換研究について（山田満稔慶應義塾大学講師提出資料） - 1421121_002.pdf
www.mext.go.jp/kaigisiryo/2019/09/__icsFiles/afieldfile/2019/09/11/1421121_002.pdf
Jan 1, 2026
41
Crystal Structure and Autoactivation Pathway of the Precursor Form of Human Tripeptidyl-peptidase 1, the Enzyme Deficient in Late Infantile Ceroid Lipofuscinosis *
www.jbc.org/article/S0021-9258(19)81813-6/fulltext
Dec 28, 2025
1
h-4065-PAR-EN - brineura-epar-public-assessment-report_en.pdf
www.ema.europa.eu/en/documents/assessment-report/brineura-epar-public-assessment-report_en.pdf
Dec 28, 2025
11
761052Orig1s000SumR.pdf
www.accessdata.fda.gov/drugsatfda_docs/nda/2017/761052Orig1s000SumR.pdf
Dec 28, 2025
11
FOCUS: an AI-assisted reading workflow for information overload | Nature Biotechnology
www.nature.com/articles/s41587-025-02947-8
Dec 20, 2025
1
Mutations in Mitochondrial Genes Ameliorate Pathology in Friedreich’s Ataxia Model
www.genengnews.com/topics/omics/mutations-in-mitochondrial-genes-ameliorate-pathology-in-friedreichs-ataxia-model/
Dec 13, 2025
33
Next-Gen CAR T Cells Destroy Cancer Cells with Fewer Toxic Effects in Mice
www.genengnews.com/topics/cancer/next-gen-car-t-cells-destroy-cancer-cells-with-fewer-toxic-effects-in-mice/
Dec 13, 2025
4
Just a moment...
www.biorxiv.org/content/10.1101/2025.11.12.687979v1.full
Dec 13, 2025
31
Neural stem cells for disease modeling and evaluation of therapeutics for infantile (CLN1/PPT1) and late infantile (CLN2/TPP1) neuronal ceroid lipofuscinoses - Orphanet Journal of Rare Diseases
link.springer.com/article/10.1186/s13023-018-0798-2
Dec 6, 2025
2
Glyco-engineered Long Acting FGF21 Variant with Optimal Pharmaceutical and Pharmacokinetic Properties to Enable Weekly to Twice Monthly Subcutaneous Dosing - Scientific Reports
www.nature.com/articles/s41598-018-22456-w
Oct 23, 2025
11
JCI - Peripherally derived FGF21 promotes remyelination in the central nervous system
www.jci.org/articles/view/94337
Oct 19, 2025
21
FGF21 Alleviates Hypoxic-Ischemic White Matter Injury in Neonatal Mice by Mediating Inflammation and Oxidative Stress Through PPAR-γ Signaling Pathway - Molecular Neurobiology
link.springer.com/article/10.1007/s12035-024-04549-y
Oct 19, 2025
1
www.reddit.com/r/ticktick/comments/1o0ao6t/can_someone_help_me_with_my_ticktick_mess/
Oct 7, 2025
2323
Immunotherapy-related cognitive impairment after CAR T cell therapy in mice
www.cell.com/cell/fulltext/S0092-8674(25)00391-5
Oct 4, 2025
11
AI Unleashed: Transforming Drug Discovery from Theory to Practice - Plenge Gen @rplenge
plengegen.com/blog/ai-sequel/
Sep 25, 2025
1
Microglia to the Rescue
www.science.org/content/blog-post/microglia-rescue
Sep 25, 2025
1
Open Targets Platform | Open Targets Platform Documentation
platform-docs.opentargets.org/
Sep 7, 2025
1