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Gain-of-Function Research

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Gain-of-function research
La ricerca di tipo "gain-of-function" migliora la comprensione dell'evoluzione virale, fornendo spunti per risposte mediche innovative a potenziali pandemie.

La ricerca di guadagno di funzione (GoFR, Gain-of-Function Research) prevede la modifica mirata di agenti biologici per alterare specifici tratti fenotipici, come la trasmissibilità, la virulenza o la gamma di ospiti. Questa disciplina impiega la genetica inversa, la mutagenesi sito-diretta e il passaggio seriale per studiare i fattori molecolari che guidano l'evoluzione virale e il passaggio zoonotico.

Identificando le mutazioni che consentono l'elusione del sistema immunitario o l'adattamento tra specie diverse, gli scienziati prevedono le minacce pandemiche e accelerano la progettazione di contromisure mediche.

Questo indice sottostante aggrega le pubblicazioni recenti sottoposte a revisione paritaria e brevetto Documenti depositati, che attestano i progressi tecnici nella genomica sintetica e nell'ingegneria virale. Queste voci delineano lo stato dei patogeni potenzialmente pandemici potenziati (ePPP) nell'ambito dei vincoli normativi relativi alla supervisione della ricerca a duplice uso e ai protocolli di biosicurezza.

Questa è la nostra ultima selezione di pubblicazioni e brevetti mondiali in inglese su Gain-of-Function, tra numerose riviste scientifiche online, classificate e focalizzate su passaggi seriali, ricerca a duplice uso di interesse, guadagno di funzione, potenziale patogeno pandemico migliorato, P3CO, miglioramento della patogenicità, miglioramento della trasmissibilità, espansione dell'ospite, tropismo virale, mutagenesi sito-diretta, genetica inversa, genomica sintetica, pressione selettiva, mutante di fuga, scheletro virale, modificazione della proteina spike, affinità di legame del recettore, potenziale di spillover zoonotico, livello di biosicurezza 4, supervisione della biosicurezza, gestione del biorischio, infezione acquisita in laboratorio, virus chimerico, salvataggio virale, inserimento del sito di clivaggio della furina, adattamento in vivo, GoFR e guadagno di funzione.

Muscle-Specific DNM2 Overexpression Improves Charcot&ndash;Marie&ndash;Tooth Disease In Vivo and Reveals a Narrow Therapeutic Window in Skeletal Muscle

Published on 2026-02-02 by Marie Goret, Gwenaelle Piccolo, Jocelyn Laporte @MDPI

Abstract: Charcot&amp;ndash;Marie&amp;ndash;Tooth disease (CMT), caused by dominant loss-of-function mutations in DNM2, encoding the GTPase dynamin-2, impairs motor and sensory function. However, the respective contributions of muscle and nerve pathology, and the therapeutic potential of increasing DNM2 expression, remain unresolved. We evaluated tissue-targeted and systemic approaches to increase DNM2 in a mouse model carrying the common K562E-CMT mutation. Muscle-specific DNM2 overexpression fro[...]


Our summary: Muscle-specific DNM2 overexpression improves symptoms in a mouse model of Charcot-Marie-Tooth disease. Systemic delivery of DNM2 worsens muscle pathology despite increased expression. Findings emphasize the importance of precise DNM2 dosage for effective treatment and highlight a narrow therapeutic window.

DNM2, Charcot-Marie-Tooth, muscle pathology, therapeutic window

Publication

Sublethal Antibiotic Exposure Induces Microevolution of Quinolone Resistance in Pathogenic Vibrio parahaemolyticus

Published on 2026-01-30 by Qian Wu, Han Yang, Tianming Xu, Pradeep K. Malakar, Huan Li, Yong Zhao @MDPI

Abstract: The microevolutionary pathways and molecular mechanisms by which the important pathogen Vibrio parahaemolyticus acquires resistance in the aquatic environment under continuous selective pressure from quinolone antibiotic residues are still unknown. Here, the study successfully simulated the long-term pressure of antibiotic residues in aquaculture by susceptible V. parahaemolyticus (VPD14) which was isolated from seafood, to a 30-day in vitro induction with sublethal concentrations of levofloxaci[...]


Our summary: Sublethal exposure to quinolone antibiotics induces microevolution in Vibrio parahaemolyticus. Mutants exhibit resistance to multiple antibiotics and adaptations such as decreased growth rates. Whole-genome sequencing reveals key mutations in resistance-determining regions associated with this resistance.

Microevolution, Quinolone Resistance, Vibrio parahaemolyticus, Antibiotic Exposure

Publication

Occurrence, Seasonal Variation, and Microbial Drivers of Antibiotic Resistance Genes in a Residential Secondary Water Supply System

Published on 2026-01-22 by Huaiyu Tian, Yu Zhou, Dawei Zhang, Weiying Li @MDPI

Abstract: The widespread use of antibiotics has led to the persistence of antibiotic-resistant bacteria (ARB) and antibiotic resistance genes (ARGs) in drinking water systems, posing potential public health risks at the point of use. In this study, a residential secondary water supply system (SWSS) in eastern China was investigated over one year to characterize microbial communities, ARB and ARG occurrence, and their associations with water quality in bulk water and biofilms. Culture-based methods, flow c[...]


Our summary: This study investigates the occurrence of antibiotic resistance genes (ARGs) in a residential secondary water supply system over one year. Significant microbial regrowth and higher ARG concentrations were observed, particularly in autumn and winter. The findings emphasize the influence of secondary distribution conditions and microbial interactions on resistance risks in drinking water systems.

Antibiotic Resistance, Microbial Communities, Water Quality, Seasonal Variation

Publication

Kr&uuml;ppel-like Factor 2 (KLF2) in the Regulation of Lipid Accumulation, ROS, and Mitochondrial Functions During Foam Cell Formation in RAW264.7 Cells

Published on 2026-01-06 by Md Sariful Islam Howlader, Manjusri Das, Surajit Hansda, Prathyusha Naidu, Hiranmoy Das @MDPI

Abstract: Foam cell formation, a hallmark of early atherosclerotic lesion development, is closely associated with mitochondrial dysfunction and excessive reactive oxygen species (ROS) production. Disruption in mitochondrial activity leads to electron leakage, elevated ROS generation, and collapse of mitochondrial membrane potential, contributing to vascular pathogenesis. In this study, we investigated the role of Kr&amp;uuml;ppel-like factor 2 (KLF2), a transcription factor known for its vasculoprotec[...]


Our summary: KLF2 regulates lipid accumulation and mitochondrial functions during foam cell formation in RAW264.7 cells. Its loss promotes foam cell formation and increases ROS, while its gain reduces these effects. This study highlights KLF2 s potential as a therapeutic target for cardiovascular diseases.

KLF2, foam cells, mitochondrial dysfunction, reactive oxygen species

Publication

Methods of treatment of neurological disorders

Patent published on the 2025-12-18 in WO under Ref WO2025259553 by PRAXIS PREC MEDICINES INC [US] (Frizzo Silvana [us], Silva De Souza Marcio [us], Petrou Steven [us])

Abstract: The present disclosure is generally directed to methods of treating a disease, disorder, or condition, e.g., a neurological disorder, a disorder associated with excessive neuronal excitability, or a disorder associated with de novo gain-of-function or loss-of-function mutations in major central nervous system sodium channel genes, such as for example, SCN1A, SCN2A, and SCN8A, using Compound 1 represented by the following structural formula:or a pharmaceutically acceptable salt thereof.[...]


Our summary: The document discusses treatment methods for neurological disorders linked to sodium channel gene mutations. It focuses on using Compound 1 or its pharmaceutically acceptable salts. The target disorders involve excessive neuronal excitability due to specific genetic mutations.

neurological disorders, sodium channel genes, treatment methods, Compound 1

Patent

Argomenti trattati: Acquisizione di funzione, evoluzione virale, risposte mediche, modificazione mirata, tratti fenotipici, trasmissibilità, virulenza, gamma di ospiti, genetica inversa, mutagenesi sito-diretta, passaggi seriali, spillover zoonotico, elusione immunitaria, adattamento interspecie, minacce pandemiche, contromisure mediche, agenti patogeni potenzialmente pandemici potenziati, supervisione della ricerca a duplice uso, protocolli di biosicurezza ISO 35001, ISO 9001, ISO 14001, ISO 45001 e ISO 17025.

Glossario dei termini utilizzati

Gain-of-Function Research (GoFR): research that involves manipulating organisms to enhance or alter their biological functions, often to study disease mechanisms, improve vaccine development, or assess potential risks of pathogens. This type of research can involve increasing transmissibility, virulence, or host range of microorganisms.

Contesto storico

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