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IGF1 as a Potential Treatment for Rett Syndrome : Safety Assessment in Six Rett Patients
Joint Authors
Morescalchi, Paolina
Bottiglioni, Ilaria
Tropea, Daniela
Scusa, Maria Flora
Benincasa, Alberto
Prina-Mello, Adriele
Borelli, Paolo
Pini, Giorgio
Di Marco, Pietro
Della-Chiesa, Andrea
Bonuccelli, Ubaldo
Congiu, Laura
Source
Issue
Vol. 2012, Issue 2012 (31 Dec. 2012), pp.1-14, 14 p.
Publisher
Hindawi Publishing Corporation
Publication Date
2012-06-13
Country of Publication
Egypt
No. of Pages
14
Main Subjects
Abstract EN
Rett syndrome (RTT) is a devastating neurodevelopmental disorder that affects one in ten thousand girls and has no cure.
The majority of RTT patients display mutations in the gene that codes for the methyl-CpG-binding protein 2 (MeCP2).
Clinical observations and neurobiological analysis of mouse models suggest that defects in the expression of MeCP2 protein compromise the development of the central nervous system, especially synaptic and circuit maturation.
Thus, agents that promote brain development and synaptic function, such as insulin-like growth factor 1 (IGF1), are good candidates for ameliorating the symptoms of RTT.
IGF1 and its active peptide, (1–3) IGF1, cross the blood brain barrier, and (1–3) IGF1 ameliorates the symptoms of RTT in a mouse model of the disease; therefore they are ideal treatments for neurodevelopmental disorders, including RTT.
We performed a pilot study to establish whether there are major risks associated with IGF1 administration in RTT patients.
Six young girls with classic RTT received IGF1 subcutaneous injections twice a day for six months, and they were regularly monitored by their primary care physicians and by the unit for RTT in Versilia Hospital (Italy).
This study shows that there are no risks associated with IGF1 administration.
American Psychological Association (APA)
Pini, Giorgio& Scusa, Maria Flora& Congiu, Laura& Benincasa, Alberto& Morescalchi, Paolina& Bottiglioni, Ilaria…[et al.]. 2012. IGF1 as a Potential Treatment for Rett Syndrome : Safety Assessment in Six Rett Patients. Autism Research and Treatment،Vol. 2012, no. 2012, pp.1-14.
https://search.emarefa.net/detail/BIM-489916
Modern Language Association (MLA)
Pini, Giorgio…[et al.]. IGF1 as a Potential Treatment for Rett Syndrome : Safety Assessment in Six Rett Patients. Autism Research and Treatment No. 2012 (2012), pp.1-14.
https://search.emarefa.net/detail/BIM-489916
American Medical Association (AMA)
Pini, Giorgio& Scusa, Maria Flora& Congiu, Laura& Benincasa, Alberto& Morescalchi, Paolina& Bottiglioni, Ilaria…[et al.]. IGF1 as a Potential Treatment for Rett Syndrome : Safety Assessment in Six Rett Patients. Autism Research and Treatment. 2012. Vol. 2012, no. 2012, pp.1-14.
https://search.emarefa.net/detail/BIM-489916
Data Type
Journal Articles
Language
English
Notes
Includes bibliographical references
Record ID
BIM-489916