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Precise insertions of large DNA fragments for cell and gene therapy.

• Authors : Li G; Gene Editing Center, School of Life Science and Technology, ShanghaiTech University, Shanghai 201210, China.; Zhang Q

Subjects: DNA*/DNA*/DNA*/genetics ; Genetic Therapy*/Genetic Therapy*/Genetic Therapy*/adverse effects; Mutagenesis, Insertional

• Source: Science bulletin [Sci Bull (Beijing)] 2023 May 15; Vol. 68 (9), pp. 857-859. Date of Electronic Publication: 2023 Apr 10.Publisher: Elsevier B.V. Country of Publication: Netherlands NLM ID: 101655530 Publication Model: Print-Electronic Cited

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Education Full Text (H.W. Wilson)

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Gene Therapy: A New Hope in Sickle Cell Disease Treatment.

• Authors : Christy, Margaret; Fisher, Beth

• Source: Journal of Pediatric Healthcare; Jan2025, Vol. 39 Issue 1, p122-129, 8p

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Pioneering the future: CRISPR-Cas9 gene therapy for hereditary hemorrhagic telangiectasia. Author's reply.

• Authors : Cerdà, Pau^1,2,3 (AUTHOR); Aguilera, Cinthia^1,3,4 (AUTHOR); Riera-Mestre, Antoni^1,2,3,5 (AUTHOR)

Subjects: *HEREDITARY hemorrhagic telangiectasia; *GENE therapy; *CRISPRS

• Source: European Journal of Internal Medicine. Sep2024, Vol. 127, p142-143. 2p.

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Pioneering the future: CRISPR-Cas9 gene therapy for hereditary hemorrhagic telangiectasia.

• Authors : James, Michael¹ (AUTHOR) ; Sehgal, Viren S.¹ (AUTHOR)

Subjects: *HEREDITARY hemorrhagic telangiectasia; *GENE therapy; *CRISPRS

• Source: European Journal of Internal Medicine. Sep2024, Vol. 127, p140-141. 2p.

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P01-11 Germline integration assessment in preclinical gene therapy studies.

• Authors : Clay, H.¹ (AUTHOR); Makori, N.² (AUTHOR); Barone, F.³ (AUTHOR)

Subjects: *GENE therapy; *GERM cells

• Source: Toxicology Letters. 2024 Supplement 2, Vol. 399, pS75-S76. 2p.

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297 Assessing the impact of lung infection on the efficacy of lentiviral vector airway gene therapy.

• Authors : McCarron, A. (AUTHOR); Reyne, N. (AUTHOR); Cmielewski, P. (AUTHOR)

Subjects: *LUNG infections; *GENE therapy

• Source: Journal of Cystic Fibrosis. Sep2024 Supplement 2, Vol. 23, pS158-S159. 2p.

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1771P Blessed: Expanded yccess for DeltaRex-G in vivo gene therapy for sarcoma, pancreas and breast cancer (NCT04091295) and other solid malignancies (IND# 19130).

• Authors : Chawla, S.P.¹ (AUTHOR); Isacoff, W.² (AUTHOR); Jeffrey, S.³ (AUTHOR)

Subjects: *PANCREATIC cancer; *GENE therapy; *BREAST cancer

• Source: Annals of Oncology. 2024 Supplement 2, Vol. 35, pS1054-S1055. 2p.

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2024 update: European consensus statement on gene therapy for spinal muscular atrophy.

• Authors : Kirschner, Janbernd; Bernert, Günther; Butoianu, Nina

Subjects: SPINAL muscular atrophy; GENE therapy; GENETIC vectors

• Source: European Journal of Paediatric Neurology; Jul2024, Vol. 51, p73-78, 6p

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M1-polarized macrophage-derived cellular nanovesicle-coated lipid nanoparticles for enhanced cancer treatment through hybridization of gene therapy and cancer immunotherapy.

• Authors : Shin, Ha Eun; Han, Jun-Hyeok; Shin, Seungyong

Subjects: GENE therapy; CANCER treatment; CANCER genes

• Source: Acta Pharmaceutica Sinica B; Jul2024, Vol. 14 Issue 7, p3169-3183, 15p

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