Search Results - "Collaud F"

Gene supplementation therapy restores motor deficits in Gnao1[C215Y] mice and proves broad efficacy in C. elegans model.

Authors: Cocozza G D'Andrea T Di Rocco M et al.

Source: Acta neuropathologica communications [Acta Neuropathol Commun] 2026 Jul 21. Date of Electronic Publication: 2026 Jul 21.

Publication Type: Journal Article

Journal Info: Publisher: BioMed Central Country of Publication: England NLM ID: 101610673 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2051-5960 (Electronic) Linking ISSN: 20515960 NLM ISO Abbreviation: Acta Neuropathol Commun Subsets: MEDLINE

Comment on: A single dose recombinant AAV based CHIKV vaccine elicits robust and durable protective antibody responses in mice.

Authors: Xu, Shenghao Li, Kaiyu Merits, Andres et al.

Source: PLoS Neglected Tropical Diseases. 11/20/2025, Vol. 19 Issue 11, p1-4. 4p.

Unremitting Neonatal Jaundice: A Diagnostic Challenge.

Authors: Sreekumari, Jyothi Nair, Sruthi Haribalakrishna, Anitha et al.

Source: Journal of Clinical Neonatology. Jan-Mar2026, Vol. 15 Issue 1, p56-60. 5p.

Novel AAV843 Vector-Mediated Gene Replacement Therapy Rescues Primary Hyperoxaluria Type I in Mice.

Authors: Zhang, Jingjia Yin, Ye Ji, Baowei et al.

Source: Cells (2073-4409). Apr2026, Vol. 15 Issue 7, p629. 20p.

Identification of alterations of immunometabolism associated with Pompe disease.

Authors: Costa-Verdera H Gargaro M Cagin U et al.

Source: Cellular and molecular life sciences : CMLS [Cell Mol Life Sci] 2026 Mar 16; Vol. 83 (1). Date of Electronic Publication: 2026 Mar 16.

Publication Type: Journal Article

Journal Info: Publisher: Springer Country of Publication: Switzerland NLM ID: 9705402 Publication Model: Electronic Cited Medium: Internet ISSN: 1420-9071 (Electronic) Linking ISSN: 1420682X NLM ISO Abbreviation: Cell Mol Life Sci Subsets: MEDLINE

Gene therapy for liver diseases: methods, challenges and opportunities.

Authors: Yan M Xiang Q

Source: Journal of nanobiotechnology [J Nanobiotechnology] 2026 May 11; Vol. 24 (1). Date of Electronic Publication: 2026 May 11.

Publication Type: Journal Article; Review

Journal Info: Publisher: BioMed Central Country of Publication: England NLM ID: 101152208 Publication Model: Electronic Cited Medium: Internet ISSN: 1477-3155 (Electronic) Linking ISSN: 14773155 NLM ISO Abbreviation: J Nanobiotechnology Subsets: MEDLINE

Application of CRISPR-Cas9-Based Gene Editing Technology in Inherited Liver Diseases.

Authors: Liu, Ran Cong, Shiqi Gao, Yuan et al.

Source: International Journal of Molecular Sciences. Jul2026, Vol. 27 Issue 14, p6469. 19p.

A Comprehensive Update on Pompe Disease: From Existing Therapies to Emerging Curative Strategies.

Authors: Estevez Barcia, Rebeca Colón, Cristóbal Hermida-Ameijeiras, Álvaro et al.

Source: International Journal of Molecular Sciences. Jul2026, Vol. 27 Issue 13, p5726. 25p.

Safety of Adeno-Associated Viral Vectors in Gene Therapy: Mechanisms of Toxicity, Clinical Risks, and Strategies for Their Minimization.

Authors: Tsaregorodtseva, Tatiana S. Radyukhina, Maria A. Ayupova, Aisylu I. et al.

Source: International Journal of Molecular Sciences. Jun2026, Vol. 27 Issue 11, p4818. 31p.

Glycogen and Glycosylation: Friends or Foes?

Authors: Konada, Rohit Sai Reddy Osborn, James Mitra, Sharmistha

Source: Biomolecules (2218-273X). Jun2026, Vol. 16 Issue 6, p885. 17p.

Emerging Technologies Tackling Adeno-Associated Viruses (AAV) Immunogenicity in Gene Therapy Applications.

Authors: Egorova, Tatiana Starikova, Anna Polikarpova, Anna

Source: Pharmaceutics. Nov2025, Vol. 17 Issue 11, p1492. 31p.

Application of Induced Pluripotent Stem Cells (iPSCs) in Hereditary and Viral Diseases of the Liver: Modeling and Treatment.

Authors: Andriianov, Vladimir Malyutina, Alina Panferov, Egor et al.

Source: International Journal of Molecular Sciences. Oct2025, Vol. 26 Issue 19, p9432. 45p.

Latest Advancements in Treatment Options for Infantile-Onset Pompe Disease: A Comprehensive Narrative Review.

Authors: Khan, Misha Ibrahim, Mufliha Riaz, Farhana et al.

Source: Clinical Medicine Insights: Pediatrics. 8/11/2025, p1-12. 12p.

ZNF146 accelerates lung adenocarcinoma progression through MDM2/p53 and PHGDH/ferroptosis.

Authors: Zhu, Junkan Ren, Shencheng Yi, Yanjun et al.

Source: Cell & Bioscience. 6/28/2025, Vol. 15 Issue 1, p1-18. 18p.

Methodological Validation and Inter-Laboratory Comparison of Microneutralization Assay for Detecting Anti-AAV9 Neutralizing Antibody in Human.

Authors: Yu, Shuangqing Zhao, Qian Zhang, Cengceng et al.

Source: Viruses (1999-4915). Oct2024, Vol. 16 Issue 10, p1512. 14p.

Pompe Disease: Pathogenesis, Molecular Mechanisms, Neurological Aspects, Diagnostics and Modern Therapeutic Approaches.

Authors: Sharshakova, Alexandra Fattakhova, Alisa Solovyeva, Valeriya et al.

Source: International Journal of Molecular Sciences. Apr2026, Vol. 27 Issue 8, p3703. 37p.

Immune Toxicities in AAV Gene Therapy: Overview for Clinicians.

Authors: Parajuli, Shestruma Gallagher, Thomas Flotte, Terence R.

Source: International Journal of Molecular Sciences. Apr2026, Vol. 27 Issue 7, p3196. 18p.

Neuromuscular Mechanisms and Oxidative Stress in Skeletal Muscle Atrophy: Emerging Stem Cell and Gene-Based Therapeutic Strategies.

Authors: Gunasekaran, Sathish Kumar Amzad Khan, Mandam Mirza, Mehwish et al.

Source: Muscles (2813-0413). Mar2026, Vol. 5 Issue 1, p13. 25p.

Editorial: Gene therapy and genome editing for metabolic liver disorders.

Authors: Collaud F Bortolussi G

Source: Frontiers in molecular medicine [Front Mol Med] 2025 Jan 09; Vol. 4, pp. 1543685. Date of Electronic Publication: 2025 Jan 09 (Print Publication: 2024).

Publication Type: Editorial

Journal Info: Publisher: Frontiers Media S.A Country of Publication: Switzerland NLM ID: 9918402378306676 Publication Model: eCollection Cited Medium: Internet ISSN: 2674-0095 (Electronic) Linking ISSN: 26740095 NLM ISO Abbreviation: Front Mol Med Subsets: PubMed not MEDLINE

A Quantitative Approach to Potency Testing for Chimeric Antigen Receptor-Encoding Lentiviral Vectors and Autologous CAR-T Cell Products, Using Flow Cytometry.

Authors: Mata-Molanes, Juan José Alserawan, Leticia España, Carolina et al.

Source: Pharmaceutics. Mar2025, Vol. 17 Issue 3, p303. 16p.