Výsledky vyhľadávania - "Gene therapy"

AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial.

Autori: Mendell JR Muntoni F McDonald CM a ďalší

Zdroj: Nature medicine [Nat Med] 2025 Jan; Vol. 31 (1), pp. 332-341. Date of Electronic Publication: 2024 Oct 09.

Spôsob vydávania: Journal Article; Randomized Controlled Trial; Clinical Trial, Phase III; Multicenter Study

Informácie o časopise: Publisher: Nature Publishing Company Country of Publication: United States NLM ID: 9502015 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1546-170X (Electronic) Linking ISSN: 10788956 NLM ISO Abbreviation: Nat Med Subsets: MEDLINE

Výrazy zo slovníka MeSH: Dependovirus*/genetics , Genetic Therapy*/methods , Muscular Dystrophy, Duchenne*/therapy , Muscular Dystrophy, Duchenne*/genetics , Gene Therapy Agents*, Adolescent ; Child ; Child, Preschool ; Humans ; Male ; Dystrophin/genetics ; Genetic Vectors/genetics ; Treatment Outcome

AAV Gene Therapy Drug Development and Translation of Engineered Ocular and Neurotropic Capsids: A Systematic Review Using Natural Language Processing.

Autori: Agbim C Wu HY Kim C a ďalší

Zdroj: Clinical and translational science [Clin Transl Sci] 2025 Dec; Vol. 18 (12), pp. e70428.

Spôsob vydávania: Journal Article; Systematic Review; Review

Informácie o časopise: Publisher: WileyBlackwell Pub Country of Publication: United States NLM ID: 101474067 Publication Model: Print Cited Medium: Internet ISSN: 1752-8062 (Electronic) Linking ISSN: 17528054 NLM ISO Abbreviation: Clin Transl Sci Subsets: MEDLINE

Výrazy zo slovníka MeSH: Dependovirus*/genetics , Capsid* , Genetic Vectors*/administration & dosage , Genetic Vectors*/genetics , Genetic Therapy*/methods , Drug Development*/methods, Humans ; Animals ; Natural Language Processing ; Translational Research, Biomedical ; Gene Therapy Agents

Key considerations for dose selection in first-in-human studies of cell and gene therapy products.

Autori: Naota M Nozaki Y Mikashima F a ďalší

Zdroj: Cytotherapy [Cytotherapy] 2025 Nov; Vol. 27 (11), pp. 1271-1278. Date of Electronic Publication: 2025 Jun 29.

Spôsob vydávania: Journal Article; Review

Informácie o časopise: Publisher: Elsevier Country of Publication: England NLM ID: 100895309 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1477-2566 (Electronic) Linking ISSN: 14653249 NLM ISO Abbreviation: Cytotherapy Subsets: MEDLINE

Výrazy zo slovníka MeSH: Genetic Therapy*/methods , Cell- and Tissue-Based Therapy*/methods, Humans ; Animals ; Gene Therapy Agents

Estimated Long-Term Durability of Valoctocogene Roxaparvovec Treatment in Male patients with Severe Hemophilia A: An Extrapolation of Clinical Data.

Autori: Santos S Robinson TM Trueman D

Zdroj: Advances in therapy [Adv Ther] 2025 Nov; Vol. 42 (11), pp. 5781-5793. Date of Electronic Publication: 2025 Sep 23.

Spôsob vydávania: Journal Article

Informácie o časopise: Publisher: Health Communications Inc Country of Publication: United States NLM ID: 8611864 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1865-8652 (Electronic) Linking ISSN: 0741238X NLM ISO Abbreviation: Adv Ther Subsets: MEDLINE

Výrazy zo slovníka MeSH: Factor VIII*/genetics , Factor VIII*/therapeutic use , Genetic Therapy*/methods , Hemophilia A*/therapy , Hemorrhage*/prevention & control, Adolescent ; Adult ; Humans ; Male ; Quality of Life ; Treatment Outcome ; Clinical Trials as Topic ; Gene Therapy Agents ; Recombinant Fusion Proteins

A proof of concept study on the use of online two-dimensional SEC-UV-RPLC-MS method for the multi-attribute characterization of gene therapy products.

Autori: Aebischer MK Rudaz S Guillarme D

Zdroj: Analytica chimica acta [Anal Chim Acta] 2025 Oct 15; Vol. 1371, pp. 344407. Date of Electronic Publication: 2025 Jul 10.

Spôsob vydávania: Journal Article

Informácie o časopise: Publisher: Elsevier Country of Publication: Netherlands NLM ID: 0370534 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1873-4324 (Electronic) Linking ISSN: 00032670 NLM ISO Abbreviation: Anal Chim Acta Subsets: MEDLINE

Výrazy zo slovníka MeSH: Dependovirus*/genetics , Dependovirus*/chemistry , Genetic Therapy* , Genetic Vectors*/genetics , Genetic Vectors*/chemistry, Chromatography, Reverse-Phase/methods ; Mass Spectrometry/methods ; Chromatography, Gel ; Proof of Concept Study ; Humans ; Gene Therapy Agents

Development of a gene therapy approach for the treatment of hereditary spastic paraplegia type 52 (SPG52)

Autori: Rodríguez Estévez, Laura

Thesis Advisors: Chillón Rodríguez, Miguel

Zdroj: TDX (Tesis Doctorals en Xarxa)

Predmety: Paraparèsia espàstica 52, Spastic paraplegia 52, Paraparesia espástica 52, Teràpia gènica, Gene therapy, Terapia génica, Malalties rares, Rare diseases, Enfermedades raras, Ciències de la Salut

Popis súboru: application/pdf

Prístupová URL adresa: http://hdl.handle.net/10803/695881

Comparative Effectiveness of Valoctocogene Roxaparvovec and Efanesoctocog Alfa in the Treatment of Severe Hemophilia A: A Matching-Adjusted Indirect Comparison of Bleeding Frequency.

Autori: Douglas TG Santos S Hinds DR a ďalší

Zdroj: Advances in therapy [Adv Ther] 2025 Nov; Vol. 42 (11), pp. 5600-5611. Date of Electronic Publication: 2025 Sep 09.

Spôsob vydávania: Comparative Study; Journal Article

Informácie o časopise: Publisher: Health Communications Inc Country of Publication: United States NLM ID: 8611864 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1865-8652 (Electronic) Linking ISSN: 0741238X NLM ISO Abbreviation: Adv Ther Subsets: MEDLINE

Výrazy zo slovníka MeSH: Factor VIII*/therapeutic use , Factor VIII*/genetics , Genetic Therapy*/methods , Hemophilia A*/therapy , Hemophilia A*/drug therapy , Hemophilia A*/complications , Hemorrhage*/prevention & control , Hemorrhage*/etiology, Adolescent ; Adult ; Child ; Female ; Humans ; Male ; Middle Aged ; Young Adult ; Treatment Outcome ; Randomized Controlled Trials as Topic ; Clinical Trials, Phase III as Topic ; Gene Therapy Agents ; Recombinant Fusion Proteins

Considerations driving the choice in clinical trial design of cell and gene therapy products: weighing convenience versus necessity.

Autori: Abrams K Aiuti A Eichler HG a ďalší

Zdroj: Cytotherapy [Cytotherapy] 2025 Oct; Vol. 27 (10), pp. 1164-1172. Date of Electronic Publication: 2025 Jun 05.

Spôsob vydávania: Journal Article

Informácie o časopise: Publisher: Elsevier Country of Publication: England NLM ID: 100895309 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1477-2566 (Electronic) Linking ISSN: 14653249 NLM ISO Abbreviation: Cytotherapy Subsets: MEDLINE

Výrazy zo slovníka MeSH: Genetic Therapy*/methods , Cell- and Tissue-Based Therapy*/methods , Research Design* , Clinical Trials as Topic*, Humans ; Randomized Controlled Trials as Topic ; Gene Therapy Agents

Validation of Guidelines for Genetic Investigation of Myeloid Neoplasms with Germline Predisposition: Results from a Prospective Cohort Study

Autori: Tesi, Bianca Robelius, Anna Baskin, Berivan a ďalší

Prispievatelia: Tesi, Bianca Robelius, Anna Baskin, Berivan a ďalší

Zdroj: Clinical Cancer Research. 31(14):3062-3071

Predmety: Medical and Health Sciences, Clinical Medicine, Cancer and Oncology, Medicin och hälsovetenskap, Klinisk medicin, Cancer och onkologi, Basic Medicine, Medical Genetics and Genomics (including Gene Therapy), Medicinska och farmaceutiska grundvetenskaper, Medicinsk genetik och genomik (Här ingår: Genterapi), Hematology, Hematologi

Prístupová URL adresa: https://doi.org/10.1158/1078-0432.CCR-24-4251

Systematic comparison of wide pore size exclusion chromatography columns for the characterization of gene therapy products.

Autori: Buff M Goyon A Elger C a ďalší

Zdroj: Journal of chromatography. A [J Chromatogr A] 2025 Jul 05; Vol. 1752, pp. 465972. Date of Electronic Publication: 2025 Apr 16.

Spôsob vydávania: Journal Article; Comparative Study

Informácie o časopise: Publisher: Elsevier Country of Publication: Netherlands NLM ID: 9318488 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1873-3778 (Electronic) Linking ISSN: 00219673 NLM ISO Abbreviation: J Chromatogr A Subsets: MEDLINE

Výrazy zo slovníka MeSH: Chromatography, Gel*/methods , Chromatography, Gel*/instrumentation , RNA, Messenger*/analysis , RNA, Messenger*/chemistry , RNA, Messenger*/isolation & purification , Genetic Therapy*, Dependovirus/genetics ; Dependovirus/chemistry ; Humans ; Porosity ; Particle Size ; RNA, Viral/analysis ; RNA, Viral/chemistry ; RNA, Viral/isolation & purification ; Gene Therapy Agents

Corticosteroid use to mitigate transaminitis-associated decline in FVIII levels following valoctocogene roxaparvovec gene therapy: clinical practice guidance.

Autori: Konkle BA Peyvandi F Foster GR a ďalší

Zdroj: Journal of thrombosis and haemostasis : JTH [J Thromb Haemost] 2025 Jul; Vol. 23 (7), pp. 2086-2094. Date of Electronic Publication: 2025 Mar 14.

Spôsob vydávania: Journal Article; Review

Informácie o časopise: Publisher: Elsevier Country of Publication: England NLM ID: 101170508 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1538-7836 (Electronic) Linking ISSN: 15387836 NLM ISO Abbreviation: J Thromb Haemost Subsets: MEDLINE

Výrazy zo slovníka MeSH: Factor VIII*/genetics , Factor VIII*/metabolism , Hemophilia A*/therapy , Hemophilia A*/blood , Hemophilia A*/genetics , Genetic Therapy*/adverse effects , Genetic Therapy*/methods , Adrenal Cortex Hormones*/therapeutic use , Adrenal Cortex Hormones*/adverse effects, Humans ; Alanine Transaminase/blood ; Adult ; Treatment Outcome ; Gene Therapy Agents ; Recombinant Fusion Proteins

Endothelial Cell pY397-FAK Expression Predicts the Risk of Breast Cancer Recurrences after Radiotherapy in the SweBCG91-RT Cohort

Autori: Drake, Rebecca J.G. Landén, Amalia H. Holmberg, Erik a ďalší

Prispievatelia: Drake, Rebecca J.G. Landén, Amalia H. Holmberg, Erik a ďalší

Zdroj: Clinical Cancer Research. 31(7):1323-1332

Predmety: Medical and Health Sciences, Clinical Medicine, Cancer and Oncology, Medicin och hälsovetenskap, Klinisk medicin, Cancer och onkologi, Basic Medicine, Medical Genetics and Genomics (including Gene Therapy), Medicinska och farmaceutiska grundvetenskaper, Medicinsk genetik och genomik (Här ingår: Genterapi)

Prístupová URL adresa: https://doi.org/10.1158/1078-0432.CCR-24-2939

Overview of Orphan Medicines in European Union: An Analysis of Regulatory and Technical-Scientific Aspects.

Autori: Santi Laurini G Nikitina V Broccoli M a ďalší

Zdroj: Therapeutic innovation & regulatory science [Ther Innov Regul Sci] 2025 Sep; Vol. 59 (5), pp. 1087-1097. Date of Electronic Publication: 2025 May 29.

Spôsob vydávania: Journal Article; Review

Informácie o časopise: Publisher: Springer International Publishing Country of Publication: Switzerland NLM ID: 101597411 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2168-4804 (Electronic) Linking ISSN: 21684790 NLM ISO Abbreviation: Ther Innov Regul Sci Subsets: MEDLINE

Výrazy zo slovníka MeSH: Orphan Drug Production*/legislation & jurisprudence , Rare Diseases*/drug therapy, European Union ; Drug Approval/legislation & jurisprudence ; Biological Products ; Gene Therapy Agents ; Time Factors ; Humans

Recombinant Adeno-Associated Virus Integration Profiles in Nonhuman Primates and Gene Therapy Participants after Treatment with Valoctocogene Roxaparvovec.

Autori: Russell CB Vettermann C Agarwal S a ďalší

Zdroj: Human gene therapy [Hum Gene Ther] 2025 Jul; Vol. 36 (13-14), pp. 945-955. Date of Electronic Publication: 2025 Jun 04.

Spôsob vydávania: Journal Article

Informácie o časopise: Publisher: M.A. Liebert Country of Publication: United States NLM ID: 9008950 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1557-7422 (Electronic) Linking ISSN: 10430342 NLM ISO Abbreviation: Hum Gene Ther Subsets: MEDLINE

Výrazy zo slovníka MeSH: Dependovirus*/genetics , Genetic Therapy*/methods , Genetic Vectors*/genetics , Genetic Vectors*/administration & dosage , Hemophilia A*/therapy , Hemophilia A*/genetics , Virus Integration*/genetics, Animals ; Humans ; Transgenes ; Primates ; Gene Therapy Agents ; Factor VIII ; Recombinant Fusion Proteins

Putative multiple myeloma susceptibility genes identified by exome sequencing of 347 familial and early-onset cases

Autori: Pertesi, Maroulio Demangel, Delphine Niroula, Abhishek a ďalší

Prispievatelia: Pertesi, Maroulio Demangel, Delphine Niroula, Abhishek a ďalší

Zdroj: Leukemia.

Predmety: Medical and Health Sciences, Clinical Medicine, Hematology, Medicin och hälsovetenskap, Klinisk medicin, Hematologi, Basic Medicine, Medical Genetics and Genomics (including Gene Therapy), Medicinska och farmaceutiska grundvetenskaper, Medicinsk genetik och genomik (Här ingår: Genterapi)

Prístupová URL adresa: https://doi.org/10.1038/s41375-025-02802-4

The genomic landscape of relapsed infant and childhood KMT2A-rearranged acute leukemia

Autori: Ahlgren, Louise Pilheden, Mattias Sturesson, Helena a ďalší

Prispievatelia: Ahlgren, Louise Pilheden, Mattias Sturesson, Helena a ďalší

Zdroj: Nature Communications. 16(1)

Predmety: Medical and Health Sciences, Basic Medicine, Medical Genetics and Genomics (including Gene Therapy), Medicin och hälsovetenskap, Medicinska och farmaceutiska grundvetenskaper, Medicinsk genetik och genomik (Här ingår: Genterapi), Clinical Medicine, Cancer and Oncology, Klinisk medicin, Cancer och onkologi, Pediatrics, Pediatrik

Prístupová URL adresa: https://doi.org/10.1038/s41467-025-64190-8

Sickle cell disease gene therapy drug expenses and reimbursement: a litmus test for commercial pricing strategy and patient access for curative therapies.

Autori: Grilley BJ Hume A Galipeau J

Zdroj: Cytotherapy [Cytotherapy] 2025 May; Vol. 27 (5), pp. 567-570. Date of Electronic Publication: 2025 Jan 23.

Spôsob vydávania: Journal Article

Informácie o časopise: Publisher: Elsevier Country of Publication: England NLM ID: 100895309 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1477-2566 (Electronic) Linking ISSN: 14653249 NLM ISO Abbreviation: Cytotherapy Subsets: MEDLINE

Výrazy zo slovníka MeSH: Anemia, Sickle Cell*/therapy , Anemia, Sickle Cell*/economics , Anemia, Sickle Cell*/genetics , Genetic Therapy*/economics , Genetic Therapy*/methods, Humans ; United States ; United States Food and Drug Administration ; Gene Therapy Agents

News.

Zdroj: Chemistry & Industry. Oct2025, Vol. 89 Issue 10, p17-21. 5p.

Predmety: GENE therapy, NEUROLOGICAL disorders, MYELIN, INVESTIGATIONAL therapies, TREATMENT effectiveness, GENETIC vectors

Preferences for Advanced Therapy Medicinal Products: Understanding the Published Literature on the Value of Innovative Health Interventions.

Autori: Ferizović, N. Lorgelly, P. Clarke, C. S. a ďalší

Zdroj: Inquiry (00469580). 11/1/2025, Vol. 62, p1-14. 14p.

Predmety: *RISK assessment, *DIFFUSION of innovations, *HEALTH insurance reimbursement, GENE therapy, MEDICAL information storage & retrieval systems, TISSUE engineering, CELLULAR therapy, DECISION making in clinical medicine, UNCERTAINTY, TREATMENT effectiveness, SYSTEMATIC reviews, MEDLINE, QUALITY of life

Empirically informed symptom severity cutoffs for body dysmorphic disorder

Autori: Mataix-Cols, David Andersson, Philip Rautio, Daniel a ďalší

Prispievatelia: Mataix-Cols, David Andersson, Philip Rautio, Daniel a ďalší

Zdroj: Journal of Psychiatric Research. 189:382-87

Predmety: Medical and Health Sciences, Basic Medicine, Medical Genetics and Genomics (including Gene Therapy), Medicin och hälsovetenskap, Medicinska och farmaceutiska grundvetenskaper, Medicinsk genetik och genomik (Här ingår: Genterapi)

Prístupová URL adresa: https://doi.org/10.1016/j.jpsychires.2025.06.019