The Use of Peptides in the Treatment of Fragile X Syndrome: Challenges and Opportunities

Fragile X Syndrome (FXS) is the most frequent cause of inherited intellectual disabilities and autism spectrum disorders, characterized by cognitive deficits and autistic behaviors. The silencing of the Fmr1 gene and consequent lack of FMRP protein, is the major contribution to FXS pathophysiology....

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Autores principales:	Alice Romagnoli, Daniele Di Marino
Formato:	article
Lenguaje:	EN
Publicado:	Frontiers Media S.A. 2021
Materias:	Fragile X syndrome targeted therapy peptides peptidomimetics drug development Psychiatry RC435-571
Acceso en línea:	https://doaj.org/article/0c86197a80b2456d8ce7f450ab063d4f
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spelling	oai:doaj.org-article:0c86197a80b2456d8ce7f450ab063d4f2021-11-04T06:11:48ZThe Use of Peptides in the Treatment of Fragile X Syndrome: Challenges and Opportunities1664-064010.3389/fpsyt.2021.754485https://doaj.org/article/0c86197a80b2456d8ce7f450ab063d4f2021-11-01T00:00:00Zhttps://www.frontiersin.org/articles/10.3389/fpsyt.2021.754485/fullhttps://doaj.org/toc/1664-0640Fragile X Syndrome (FXS) is the most frequent cause of inherited intellectual disabilities and autism spectrum disorders, characterized by cognitive deficits and autistic behaviors. The silencing of the Fmr1 gene and consequent lack of FMRP protein, is the major contribution to FXS pathophysiology. FMRP is an RNA binding protein involved in the maturation and plasticity of synapses and its absence culminates in a range of morphological, synaptic and behavioral phenotypes. Currently, there are no approved medications for the treatment of FXS, with the approaches under study being fairly specific and unsatisfying in human trials. Here we propose peptides/peptidomimetics as candidates in the pharmacotherapy of FXS; in the last years this class of molecules has catalyzed the attention of pharmaceutical research, being highly selective and well-tolerated. Thanks to their ability to target protein-protein interactions (PPIs), they are already being tested for a wide range of diseases, including cancer, diabetes, inflammation, Alzheimer's disease, but this approach has never been applied to FXS. As FXS is at the forefront of efforts to develop new drugs and approaches, we discuss opportunities, challenges and potential issues of peptides/peptidomimetics in FXS drug design and development.Alice RomagnoliDaniele Di MarinoFrontiers Media S.A.articleFragile X syndrometargeted therapypeptidespeptidomimeticsdrug developmentPsychiatryRC435-571ENFrontiers in Psychiatry, Vol 12 (2021)
institution	DOAJ
collection	DOAJ
language	EN
topic	Fragile X syndrome targeted therapy peptides peptidomimetics drug development Psychiatry RC435-571
spellingShingle	Fragile X syndrome targeted therapy peptides peptidomimetics drug development Psychiatry RC435-571 Alice Romagnoli Daniele Di Marino The Use of Peptides in the Treatment of Fragile X Syndrome: Challenges and Opportunities
description	Fragile X Syndrome (FXS) is the most frequent cause of inherited intellectual disabilities and autism spectrum disorders, characterized by cognitive deficits and autistic behaviors. The silencing of the Fmr1 gene and consequent lack of FMRP protein, is the major contribution to FXS pathophysiology. FMRP is an RNA binding protein involved in the maturation and plasticity of synapses and its absence culminates in a range of morphological, synaptic and behavioral phenotypes. Currently, there are no approved medications for the treatment of FXS, with the approaches under study being fairly specific and unsatisfying in human trials. Here we propose peptides/peptidomimetics as candidates in the pharmacotherapy of FXS; in the last years this class of molecules has catalyzed the attention of pharmaceutical research, being highly selective and well-tolerated. Thanks to their ability to target protein-protein interactions (PPIs), they are already being tested for a wide range of diseases, including cancer, diabetes, inflammation, Alzheimer's disease, but this approach has never been applied to FXS. As FXS is at the forefront of efforts to develop new drugs and approaches, we discuss opportunities, challenges and potential issues of peptides/peptidomimetics in FXS drug design and development.
format	article
author	Alice Romagnoli Daniele Di Marino
author_facet	Alice Romagnoli Daniele Di Marino
author_sort	Alice Romagnoli
title	The Use of Peptides in the Treatment of Fragile X Syndrome: Challenges and Opportunities
title_short	The Use of Peptides in the Treatment of Fragile X Syndrome: Challenges and Opportunities
title_full	The Use of Peptides in the Treatment of Fragile X Syndrome: Challenges and Opportunities
title_fullStr	The Use of Peptides in the Treatment of Fragile X Syndrome: Challenges and Opportunities
title_full_unstemmed	The Use of Peptides in the Treatment of Fragile X Syndrome: Challenges and Opportunities
title_sort	use of peptides in the treatment of fragile x syndrome: challenges and opportunities
publisher	Frontiers Media S.A.
publishDate	2021
url	https://doaj.org/article/0c86197a80b2456d8ce7f450ab063d4f
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The Use of Peptides in the Treatment of Fragile X Syndrome: Challenges and Opportunities

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