Search Results

Extensive rod and cone photoreceptor-cell degeneration in rat models of giant axonal neuropathy: implications for gene therapy of human disease.

• Authors : Armao, Diane^1,2 (AUTHOR) ; Bouldin, Thomas W.¹ (AUTHOR); Bailey, Rachel M.³ (AUTHOR)

Subjects: *PHOTORECEPTORS; *GENE therapy; *SOCIAL degeneration

• Source: Ophthalmic Genetics. Oct2021, Vol. 42 Issue 5, p600-603. 4p.

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Algae allow blind mice to see the light.

• Authors : Hooper, Rowan (AUTHOR)

Subjects: *GENE therapy; *ALGAE; *RETINA

• Source: New Scientist. 4/16/2011, Vol. 210 Issue 2808, p10-11. 2p.

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Discovering human cell-compatible gene therapy virus variants via optimized screening in mouse models.

• Authors : Moyu Dai; Ning Yang; Kai Xu

Subjects: GENE therapy; LABORATORY mice; HUMAN genes

• Source: Cell Proliferation. Mar2024, Vol. 57 Issue 3, p1-11. 11p.

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In utero gene therapy rescues microcephaly caused by Pqbp1-hypofunction in neural stem progenitor cells.

• Authors : Ito, H; Shiwaku, H; Yoshida, C

Subjects: GENE therapy; MICROCEPHALY; NEURAL stem cells

• Source: Molecular Psychiatry. Apr2015, Vol. 20 Issue 4, p459-471. 13p.

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Gene therapy in Aβ-induced cell and mouse models of Alzheimer's disease through compensating defective mitochondrial complex I function.

• Authors : Li, Hongzhi; Chen, Zhuo; Shen, Yuqi

Subjects: ALZHEIMER'S disease; HIPPOCAMPUS (Brain); GENE therapy

• Source: Journal of Translational Medicine; 8/14/2024, Vol. 22 Issue 1, p1-16, 16p

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Partial correction of immunodeficiency by lentiviral vector gene therapy in mouse models carrying Rag1 hypomorphic mutations.

• Authors : Castiello, Maria Carmina; Di Verniere, Martina; Draghici, Elena

Subjects: SEVERE combined immunodeficiency; GENE therapy; TALL-1 (Protein)

• Source: Frontiers in Immunology; 2023, p1-19, 19p

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Magnet enhances gene therapy for cancer.

Subjects: GENE therapy; MAGNETS; CANCER treatmentENGLANDENGLANDENGLANDReport Available

• Source: New Scientist. 4/26/2008, Vol. 198 Issue 2653, p23-23. 1p.

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A Mouse Model of X-Linked Chronic Granulomatous Disease for the Development of CRISPR/Cas9 Gene Therapy.

• Authors : Sevim-Wunderlich, Seren; Dang, Tu; Rossius, Jana

Subjects: CHRONIC granulomatous disease; GENE therapy; PRIMARY immunodeficiency diseases

• Source: Genes; Jun2024, Vol. 15 Issue 6, p706, 17p

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CLN7 gene therapy: hope for an ultra-rare condition.

• Authors : Brudvig, Jon J.¹; Weimer, Jill M.¹

Subjects: *GENE therapy; *GLYCOGEN storage disease type II; *NEURONAL ceroid-lipofuscinosis

• Source: Journal of Clinical Investigation. Mar2022, Vol. 132 Issue 5, p1-3. 3p.

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Paving a way to treat spastic paraplegia 50.

• Authors : Brent, Jonathan R.¹; Han-Xiang Deng¹

Subjects: *PARAPLEGIA; *GENE therapy; *PAVEMENTS

• Source: Journal of Clinical Investigation. 5/15/2023, Vol. 133 Issue 10, p1-3. 3p.

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