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In most main group element compounds, the valence electrons of the isolated atoms combine to form chemical bonds that satisfy the octet rule. For instance, the four valence electrons of carbon overlap with electrons from four hydrogen atoms to form CH4. The one valence electron leaves sodium and adds to the seven valence electrons of chlorine to form the ionic formula unit NaCl (Figure 1a). Transition metals do not normally bond in this fashion. They primarily form coordinate covalent bonds, a...
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The structure of a crystalline solid, whether a metal or not, is best described by considering its simplest repeating unit, which is referred to as its unit cell. The unit cell consists of lattice points that represent the locations of atoms or ions. The entire structure then consists of this unit cell repeating in three dimensions. The three different types of unit cells present in the cubic lattice are illustrated in Figure 1.
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A Coordinated Attack: Rett Syndrome Therapeutic Development.

Rocco G Gogliotti1, Colleen M Niswender2

  • 1Vanderbilt Department of Pharmacology and Vanderbilt Center for Neuroscience Drug Discovery, Vanderbilt University, Nashville, TN, USA.

Trends in Pharmacological Sciences
|March 26, 2019
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Summary
This summary is machine-generated.

Rett syndrome (RTT) is a neurodevelopmental disorder linked to the MeCP2 gene. This article explores various drug strategies and preclinical study designs for effective RTT treatment.

Keywords:
Rett syndromepharmacological intervention

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Area of Science:

  • Neuroscience
  • Genetics
  • Pharmacology

Background:

  • Rett syndrome (RTT) is a significant neurodevelopmental disorder.
  • Mutations in the Methyl CpG binding protein 2 (MeCP2) gene are the primary cause of RTT.
  • Current treatment options for RTT are limited, necessitating novel therapeutic approaches.

Purpose of the Study:

  • To review and discuss current and emerging pharmacological strategies for treating Rett syndrome.
  • To explore the potential of drug repurposing and de novo drug discovery for RTT.
  • To analyze the importance of preclinical study design and outcome measures in advancing RTT therapeutics.

Main Methods:

  • Literature review of preclinical and clinical studies on RTT.
  • Analysis of drug repurposing initiatives targeting MeCP2-related pathways.
  • Evaluation of de novo drug discovery pipelines for RTT.

Main Results:

  • Multiple pharmacological strategies are under investigation for RTT.
  • Drug repurposing shows promise by identifying existing drugs that may benefit RTT patients.
  • De novo discovery efforts are exploring novel compounds to address the underlying pathology of RTT.

Conclusions:

  • A multifaceted pharmacological approach is crucial for effective Rett syndrome treatment.
  • Optimizing preclinical study design and outcome measures is essential for successful translation to clinical practice.
  • Continued research into both drug repurposing and de novo discovery holds significant potential for improving RTT outcomes.