Mariana Bustamante

Enabling endpoint development for interventional clinical trials in individuals with Angelman syndrome: a prospective, longitudinal, observational clinical study (FREESIAS)

Jorrit Tjeertes, Carlos A. Bacino, Terry Jo Bichell, Lynne M. Bird et autres

BACKGROUND: Angelman syndrome (AS) is a rare neurodevelopmental disorder characterized by the absence of a functional UBE3A gene, which causes developmental, behavioral, and medical challenges. While currently untreatable, comprehensive data could help identify appropriate endpoints assessing meaningful improvements in clinical trials. Herein …

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A first-in-human study of the fibroblast activation protein–targeted, 4-1BB agonist RO7122290 in patients with advanced solid tumors

Ignacio Melero, Tamara Tanos, Mariana Bustamante, Miguel Fernandez de Sanmamed et autres

This first-in-human study evaluated RO7122290, a bispecific fusion protein carrying a split trimeric 4-1BB (CD137) ligand and a fibroblast activation protein α (FAP) binding site that costimulates T cells for improved tumor cell killing in FAP-expressing tumors. Patients with advanced or metastatic …

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Enabling endpoint development for interventional clinical trials in individuals with Angelman syndrome: a prospective, longitudinal, observational clinical study (FREESIAS)

Jorrit Tjeertes, Carlos A. Bacino, Terry Jo Bichell, Lynne M. Bird et autres

Abstract Background: Angelman syndrome (AS) is a rare neurodevelopmental disorder with developmental, behavioral, and medical challenges caused by absence of functional UBE3A. AS has unmet treatment needs and no approved therapies address its underlying pathophysiology. To develop new therapies, comprehensive data are …

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Gene Expression Analysis by Quantitative Real-Time PCR for Floral Tissues

Raquel Álvarez-Urdiola, Mariana Bustamante, Joana Ribes, José Luis Riechmann

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527 Digital spatial profiling of paired tumor biopsies reveals indoleamine 2,3-dioxygenase (IDO)1 as a potential resistance mechanism for a tumor-targeted 4–1BB agonist in patients with solid tumors

Tamara Tanos, Christian Heichinger, Sabine Wilson, Marta Cañamero et autres

Background We previously described the capacity of RO7122290 (RO) - a FAP-targeted 4-1BB bispecific antibody - to induce CD8+ T cell infiltration and activation in the tumor (Moreno V. et al, SITC 2020). Aiming to compare pharmacodynamic (PD) changes in tumor nests …

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Clinical Characterization of Epilepsy in Children With Angelman Syndrome

Daiana Cassater, Mariana Bustamante, Lisa Sach-Peltason, Alexander Rotenberg et autres

BACKGROUND: Epilepsy is highly prevalent in children with Angelman syndrome (AS), and its detailed characterization and relationship to the genotype (deletion vs nondeletion) is important both for medical practice and for clinical trial design. METHODS AND MATERIALS: We retrospectively analyzed the main …

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Overexpression of the vascular brassinosteroid receptor BRL3 confers drought resistance without penalizing plant growth

Norma Fàbregas, Fidel Lozano‐Elena, David Blasco‐Escámez, Takayuki Tohge et autres

Drought represents a major threat to food security. Mechanistic data describing plant responses to drought have been studied extensively and genes conferring drought resistance have been introduced into crop plants. However, plants with enhanced drought resistance usually display lower growth, highlighting the …

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Genome-wide analyses for dissecting gene regulatory networks in the shoot apical meristem

Mariana Bustamante, José Tomás Matus, José Luis Riechmann

Shoot apical meristem activity is controlled by complex regulatory networks in which components such as transcription factors, miRNAs, small peptides, hormones, enzymes and epigenetic marks all participate. Many key genes that determine the inherent characteristics of the shoot apical meristem have been …

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Gene Expression Analysis by Quantitative Real-time PCR for Floral Tissues

Mariana Bustamante, Jian Jin, Oriol Casagran, Tania Nolan et autres

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