The AID2 system offers a potent tool for rapid, reversible, or sustained degradation of essential proteins in live mice DOI Creative Commons
Valentina C. Sladky, Margaret A. Strong, Daniel Tapias-Gomez

и другие.

bioRxiv (Cold Spring Harbor Laboratory), Год журнала: 2024, Номер unknown

Опубликована: Июнь 5, 2024

Abstract Studying essential genes required for dynamic processes in live mice is challenging as genetic perturbations are irreversible and limited by slow protein depletion kinetics. The first-generation auxin-inducible-degron (AID) system a powerful tool analyzing inducible loss cultured cells. However, auxin administration toxic to mice, preventing its long-term use animals. Here, we an optimized second-generation AID achieve the conditional reversible of centrosomal CEP192 mice. We show that derivative 5-Ph-IAA well tolerated over two weeks drives near-complete CEP192-mAID degradation less than one hour vivo . Prolonged led cell division failure death proliferative tissues. Thus, suited rapid and/or sustained offering valuable new interrogating function

Язык: Английский

Degron tagging for rapid protein degradation in mice DOI Creative Commons
Brianda Areli Hernández-Morán,

Gillian C.A. Taylor,

Álvaro Lorente‐Macías

и другие.

Disease Models & Mechanisms, Год журнала: 2024, Номер 17(4)

Опубликована: Апрель 1, 2024

Degron tagging allows proteins of interest to be rapidly degraded, in a reversible and tuneable manner, response chemical stimulus. This provides numerous opportunities for understanding disease mechanisms, modelling therapeutic interventions constructing synthetic gene networks. In recent years, many laboratories have applied degron successfully cultured mammalian cells, spurred by rapid advances the fields genome editing targeted protein degradation. this At Glance article, we focus on efforts apply mouse models, discussing distinct set challenges posed vivo environment.

Язык: Английский

Процитировано

5

PROTAC-Based Protein Degradation as a Promising Strategy for Targeted Therapy in Sarcomas DOI Open Access
Caterina Mancarella, Andrea Morrione, Katia Scotlandi

и другие.

International Journal of Molecular Sciences, Год журнала: 2023, Номер 24(22), С. 16346 - 16346

Опубликована: Ноя. 15, 2023

Sarcomas are heterogeneous bone and soft tissue cancers representing the second most common tumor type in children adolescents. Histology genetic profiling discovered more than 100 subtypes, which characterized by peculiar molecular vulnerabilities. However, limited therapeutic options exist beyond standard therapy clinical benefits from targeted therapies were observed only a minority of patients with sarcomas. The rarity these tumors, paucity actionable mutations, limitations chemical composition current hindered use approaches Targeted protein degradation (TPD) is an innovative pharmacological modality to directly alter abundance promising potential cancer, even for undruggable proteins. TPD based on small molecules called degraders or proteolysis-targeting chimeras (PROTACs), trigger ubiquitin-dependent interest. In this review, we will discuss major features PROTAC PROTAC-derived systems target validation cancer treatment focus overcome issues connected sarcomas, including drug resistance, specificity, targets. A deeper understanding strategies might provide new fuel drive personalized medicine

Язык: Английский

Процитировано

9

Differential Protein Degradation with a Super-Degron Tag and Thalidomide Analogs: EGFP is Degraded in Wild-Type Mouse Cell, While PD-1 Requires CRBN Humanization, Providing New Insights into T Cell Regulation DOI
Chie Naruse, Osamu Ishibashi, Masatoshi Ohgushi

и другие.

Опубликована: Янв. 1, 2025

It was previously considered that protein knockdown using a combination of zinc finger degron tag and thalidomide analog is impossible in mouse cells, however, EGFP expression as an indicator, we found possible super-degron (SD) with iberdomide or mezigdomide cells. Despite the efficient degradation SD-tagged wild-type PD-1 could not be degraded but human Jurkat cells human-type CEREBLON (CRBNI391V). In mice CRBNI391V, endogenous tagged SD efficiently suppressed T both cultured whole body. addition, comparison treatment anti-PD-1 antibody suggested transient downregulation might prevent cell exhaustion compared to treatment. brain also reduced by pomalidomide crosses brain-blood barrier. These results suggest analogs are effective for vitro vivo knockdown, although some conditional settings required.

Язык: Английский

Процитировано

0

Rapid and sustained degradation of the essential centrosome protein CEP192 in live mice using the AID2 system DOI Creative Commons
Valentina C. Sladky, Margaret A. Strong, Daniel Tapias-Gomez

и другие.

Science Advances, Год журнала: 2025, Номер 11(9)

Опубликована: Фев. 28, 2025

Studying essential genes required for dynamic processes in live mice is challenging as genetic perturbations are irreversible and limited by slow protein depletion kinetics. The auxin-inducible degron (AID) system a powerful tool analyzing inducible loss vitro, but it toxic to mice. Here, we use an optimized second-generation AID achieve the conditional reversible of centrosomal CEP192 We show that auxin derivative 5-phenyl-indole-3-acetic acid well tolerated over 2 weeks drives near-complete degradation less than 1 hour vivo. did not affect centriole duplication decreased γ-tubulin recruitment centrosomes impairing mitotic spindle assembly. Sustained vivo led cell division failure death proliferative tissues. Thus, suited rapid and/or sustained study functions

Язык: Английский

Процитировано

0

Drug discovery technologies–Current and future trends DOI

Mark C. Noe,

Claire M. Steppan,

Andrea D. Weston

и другие.

Elsevier eBooks, Год журнала: 2025, Номер unknown

Опубликована: Янв. 1, 2025

Язык: Английский

Процитировано

0

Establishment and characterization of mouse lines useful for endogenous protein degradation via an improved auxin‐inducible degron system (AID2) DOI Creative Commons

Hatsune Makino‐Itou,

Noriko Yamatani,

Akemi Okubo

и другие.

Development Growth & Differentiation, Год журнала: 2024, Номер 66(7), С. 384 - 393

Опубликована: Сен. 1, 2024

Abstract The development of new technologies opens avenues in the research field. Gene knockout is a key method for analyzing gene function mice. Currently, conditional strategies are employed to examine temporal and spatial function. However, phenotypes sometimes not observed because time required depletion due long half‐life target proteins. Protein knockdown using an improved auxin‐inducible degron system, AID2, overcomes such difficulties owing rapid efficient depletion. We AID‐tagged proteins within few several hours by simple intraperitoneal injection auxin analog, 5‐Ph‐IAA, which much shorter than knockout. Importantly, loss protein reversible, making useful measure effects transient Here, we also established mouse lines AID2‐medicated knockdown, include knock‐in ROSA26 locus; one expresses TIR1(F74G), other reporter expressing AID‐mCherry. germ‐cell‐specific TIR1 line confirmed specificity. In addition, introduced AID tag endogenous protein, DCP2 via CAS9‐mediated editing method. that was effectively eliminated resulted similar phenotype 20 h.

Язык: Английский

Процитировано

1

The AID2 system offers a potent tool for rapid, reversible, or sustained degradation of essential proteins in live mice DOI Creative Commons
Valentina C. Sladky, Margaret A. Strong, Daniel Tapias-Gomez

и другие.

bioRxiv (Cold Spring Harbor Laboratory), Год журнала: 2024, Номер unknown

Опубликована: Июнь 5, 2024

Abstract Studying essential genes required for dynamic processes in live mice is challenging as genetic perturbations are irreversible and limited by slow protein depletion kinetics. The first-generation auxin-inducible-degron (AID) system a powerful tool analyzing inducible loss cultured cells. However, auxin administration toxic to mice, preventing its long-term use animals. Here, we an optimized second-generation AID achieve the conditional reversible of centrosomal CEP192 mice. We show that derivative 5-Ph-IAA well tolerated over two weeks drives near-complete CEP192-mAID degradation less than one hour vivo . Prolonged led cell division failure death proliferative tissues. Thus, suited rapid and/or sustained offering valuable new interrogating function

Язык: Английский

Процитировано

0