Tento výsledek nelze pro nepřihlášené uživatele zobrazit.
K zobrazení výsledku je třeba se přihlásit.

CARDIOVASCULAR DISEASE, SIGNALING, GENE/CELL THERAPY AND ADVANCED NANOBIOMATERIALS.

Autor: Erdil, Nevzat¹ nevzat.erdil@inonu.edu.tr

Publikováno v: Advances in Biology & Earth Sciences. 2024 Special Issue, Vol. 9, p58-80. 23p.

Therapeutic advantages of combined gene/cell therapy strategies in a murine model of GM2 gangliosidosis

Autor: Davide Sala, Francesca Ornaghi, Francesco Morena, Chiara Argentati, Manuela Valsecchi, Valeria Alberizzi, Roberta Di Guardo, Alessandra Bolino, Massimo Aureli, Sabata Martino, Angela Gritti

Publikováno v: Molecular Therapy: Methods & Clinical Development, Vol 25, Iss , Pp 170-189 (2022)

Genetic deficiency of β-N-acetylhexosaminidase (Hex) functionality leads to accumulation of GM2 ganglioside in Tay-Sachs disease and Sandhoff disease (SD), which presently lack approved therapies. Current experimental gene therapy (GT) approaches wi

Externí odkaz: https://doaj.org/article/61bdd9f6fc0d43f78a06d041a2b5f2f0

Tento výsledek nelze pro nepřihlášené uživatele zobrazit.
K zobrazení výsledku je třeba se přihlásit.

2022 White Paper on Recent Issues in Bioanalysis: ICH M10 BMV Guideline & Global Harmonization; Hybrid Assays; Oligonucleotides & ADC; Non-Liquid & Rare Matrices; Regulatory Inputs ( Part 1A - Recommendations on Mass Spectrometry, Chromatography and Sample Preparation, Novel Technologies, Novel Modalities, and Novel Challenges, ICH M10 BMV Guideline & Global Harmonization Part 1B - Regulatory Agencies' Inputs on Regulated Bioanalysis/BMV, Biomarkers/CDx/BAV, Immunogenicity, Gene & Cell Therapy and Vaccine).

Autor: Szapacs M; Abbvie, Philadelphia, PA, USA., Jian W; Janssen, Spring House, PA, USA., Spellman D; Merck, West Point, PA, USA., Cunliffe J; Novartis, Cambridge, MA, USA., Verburg E; Lilly, Indianapolis, IN, USA., Kaur S; Genentech, South San Francisco, CA, USA., Kellie J; GlaxoSmithKline, Collegeville, PA, USA., Li W; Novartis, East Hanover, NJ, USA., Mehl J; GlaxoSmithKline, Collegeville, PA, USA., Qian M; Takeda, Cambridge, MA, USA., Qiu X; WRIB, Montreal, QC, Canada., Sirtori FR; EMD Serono, Ivrea, TO, Italy., Rosenbaum AI; AstraZeneca, South San Francisco, CA, USA., Sikorski T; GlaxoSmithKline, Collegeville, PA, USA., Surapaneni S; Bristol-Myers Squibb, Lawrenceville, NJ, USA., Wang J; Crinetics, San Diego, CA, USA., Wilson A; AstraZeneca, Cambridge, UK., Zhang J; US FDA, Silver Spring, MD, USA., Xue Y; Bristol-Myers Squibb, Lawrenceville, NJ, USA., Post N; Ionis, Carlsbad, CA, USA., Huang Y; AstraZeneca, South San Francisco, CA, USA., Goykhman D; Merck, West Point, PA, USA., Yuan L; Biogen, Cambridge, MA, USA., Fang K; GlaxoSmithKline, Collegeville, PA, USA., Casavant E; Genentech, South San Francisco, CA, USA., Chen L; Boehringer Ingelheim, Ridgefield, CT, USA., Fu Y; Novartis, East Hanover, NJ, USA., Huang M; Regeneron, Tarrytown, NY, USA., Ji A; Chiesi, Boston, MA, USA., Johnson J; Pfizer, Andover, MA, USA., Lassman M; Merck, West Point, PA, USA., Li J; Alnylam, Cambridge, MA, USA., Saad O; Genentech, South San Francisco, CA, USA., Sarvaiya H; Abbvie, San Mateo, CA, USA., Tao L; Sanofi, Framingham, MA, USA., Wang Y; WRIB, Montreal, QC, Canada., Zheng N; Bristol-Myers Squibb, Lawrenceville, NJ, USA., Dasgupta A; US FDA, Silver Spring, MD, USA., Abhari MR; US FDA, Silver Spring, MD, USA., Ishii-Watabe A; Japan MHLW, Tokyo, Japan., Saito Y; Japan MHLW, Tokyo, Japan., Mendes Fernandes DN; Brazil ANVISA, Brasilia, Brazil., Bower J; Precision for Medicine, Chantilly, VA, USA., Burns C; UK MHRA, London, UK., Carleton K; Janssen, Spring House, PA, USA., Cho SJ; US FDA, Silver Spring, MD, USA., Du X; US FDA, Silver Spring, MD, USA., Fjording M; EWG ICH M10, Copenhagen, Denmark., Garofolo F; BRI Frontage, Vancouver, BC, Canada., Kar S; WRIB, Montreal, QC, Canada., Kavetska O; Pfizer, Groton, CT, USA., Kossary E; WHO, Geneva, Switzerland., Lu Y; US FDA, Silver Spring, MD, USA., Mayer A; GlaxoSmithKline, Collegeville, PA, USA., Palackal N; Regeneron, Tarrytown, NY, USA., Salha D; Altasciences, Laval, QC, Canada., Thomas E; Labcorp, Indianapolis, IN, USA., Verhaeghe T; Janssen, Beerse, Belgium., Vinter S; UK MHRA, London, UK., Wan K; Pfizer, San Diego, CA, USA., Wang YM; US FDA, Silver Spring, MD, USA., Williams K; Genentech, South San Francisco, CA, USA., Woolf E; Merck, West Point, PA, USA., Yang L; US FDA, Silver Spring, MD, USA., Yang E; GlaxoSmithKline, Collegeville, PA, USA., Bandukwala A; US FDA, Silver Spring, MD, USA., Hopper S; UK MHRA, London, UK., Maher K; US FDA, Silver Spring, MD, USA., Xu J; US FDA, Jefferson, AR, USA., Brodsky E; US FDA, Silver Spring, MD, USA., Cludts I; UK MHRA, London, UK., Irwin C; Health Canada, Ottawa, ON, Canada., Joseph J; Health Canada, Ottawa, ON, Canada., Kirshner S; US FDA, Silver Spring, MD, USA., Manangeeswaran M; US FDA, Silver Spring, MD, USA., Maxfield K; US FDA, Silver Spring, MD, USA., Pedras-Vasconcelos J; US FDA, Silver Spring, MD, USA., Solstad T; Norway NoMA, Oslo, Norway., Thacker S; US FDA, Silver Spring, MD, USA., Tounekti O; Health Canada, Ottawa, ON, Canada., Verthelyi D; US FDA, Silver Spring, MD, USA., Wadhwa M; UK MHRA, London, UK., Wagner L; US FDA, Silver Spring, MD, USA., Yamamoto T; Japan MHLW, Tokyo, Japan., Zhang L; Health Canada, Ottawa, ON, Canada., Zhou L; US FDA, Silver Spring, MD, USA.

Publikováno v: Bioanalysis [Bioanalysis] 2023 Aug; Vol. 15 (16), pp. 955-1016. Date of Electronic Publication: 2023 Aug 31.