Neurofibromin: Difference between revisions

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Glutamine 61 of Ras is a residue that facilitates the conversion of GTP to GDP, turning Ras from its active state to inactive state. There is a catalytic water molecule that glutamine interacts with to position the molecule for a nucleophilic attack on the gamma phosphate of GTP. Mutations of this residue have been related to lower rates of hydrolysis. <ref name= ''Frech''>PMID:8136358</ref>. Tyrosine 32 makes water-mediated hydrogen bonds with the gamma phosphate of GTP. This position is also where Ras is phosphorylation to promote the activity of GTPase-activating proteins and GTP hydrolysis. <ref name= ''Bunda''>DOI:10.1038/ncomms9859</ref>  
Glutamine 61 of Ras is a residue that facilitates the conversion of GTP to GDP, turning Ras from its active state to inactive state. There is a catalytic water molecule that glutamine interacts with to position the molecule for a nucleophilic attack on the gamma phosphate of GTP. Mutations of this residue have been related to lower rates of hydrolysis. <ref name= ''Frech''>PMID:8136358</ref>. Tyrosine 32 makes water-mediated hydrogen bonds with the gamma phosphate of GTP. This position is also where Ras is phosphorylation to promote the activity of GTPase-activating proteins and GTP hydrolysis. <ref name= ''Bunda''>DOI:10.1038/ncomms9859</ref>  
==RAS Complex==  
==RAS Complex==  
== Disease Relevance ==
Mutations to the neurofibromin protein are implicated in the progression of Neurofibromatosis type 1. Neurofibromatosis type 1 is a condition where cancer develops by inactivating the Ras suppression effects of NF, allowing Ras to behave as an oncogene. Neurofibromatosis type 1 is an autosomal dominant disorder that affects 1 in 3,000 people. The ''NF'' gene has the highest mutation rate of any human gene, adding to the prevalence of cancers related to neurofibromin mutations<ref name= ''Lupton''>PMID:34887559</ref>. Furthermore, these mutations consist heavily of ''de novo'' mutations<ref name= ''Abramowicz''>PMID:25182393</ref>. NF1 primarily causes tumors in the central and peripheral nervous systems, but often has a multisystem expression including tumors in the dermatologic, cardiovascular, gastrointestinal, and orthopedic systems<ref name= ''Cimino''>PMID:29478615</ref>. The wide range of presentations is consistent with the multiplicity of mutations observed in the causative protein<ref name= ''Ly''>PMID:31582003</ref>. This multiplicity derives from the immense size and homo dimeric nature of the neurofibromin protein that allows for otherwise innocuous mutations to wreak havoc on the conformations of the protein as well as its ability to bind to the Ras protein as positioning of NF is highly important.


===Downstream Effects===
===Downstream Effects===
[[Image:Signal_transduction_pathways.png|600 px|right|thumb|Figure 2: Downstream proliferation of Ras signals have a variety of impacts, including gene regulation, cell proliferation, and cell growth; By cybertory - This file was derived from: Signal transduction v1.png, CC BY-SA 3.0, https://commons.wikimedia.org/w/index.php?curid=12081090]]
[[Image:Signal_transduction_pathways.png|600 px|right|thumb|Figure 2: Downstream proliferation of Ras signals have a variety of impacts, including gene regulation, cell proliferation, and cell growth; By cybertory - This file was derived from: Signal transduction v1.png, CC BY-SA 3.0, https://commons.wikimedia.org/w/index.php?curid=12081090]]
The NF1 gene that neurofibromin encodes has a high mutation rate, however it is difficult to detect the mutations because of how large the protein is and how randomly the mutations are distributed across the protein. Many of the mutations have been detected in the catalytic Gap-related domain. Misregulated [http://https://proteopedia.org/wiki/index.php/GTPase_HRas Ras activity] can lead to uncontrolled signaling in many different cell signaling pathways. Figure 2 provides an overview of the pathways that are connected to Ras, such as the MEK pathway. Eventually, the pathways relate to gene regulation and cell proliferation. If Ras is hyperactive, cell overgrowth and cancer can result. A focus of drug design is the suppression of Ras and its downstream signaling pathways.
Misregulated [http://https://proteopedia.org/wiki/index.php/GTPase_HRas Ras activity] can lead to uncontrolled signaling in many different cell signaling pathways. Figure 2 provides an overview of the pathways that are connected to Ras, such as the MEK pathway.  
 
== Disease Relevance ==
Mutations to the neurofibromin protein are implicated in the progression of Neurofibromatosis type 1 (NF1). This condition drives several forms of human cancers by inactivating the Ras suppression effects of NF, allowing Ras to behave as an oncogene. Neurofibromatosis type 1 is an autosomal dominant disorder that affects 1 in 3,000 people, and the NF gene itself has the highest mutation rate of any known human gene, adding to its prevalence<ref name= ''Lupton''>PMID:34887559</ref>. Furthermore, these mutations consist heavily of ''de novo'' mutations<ref name= ''Abramowicz''>PMID:25182393</ref>. NF1 primarily causes tumors in the central and peripheral nervous systems, but often has a multisystem expression including tumors in the dermatologic, cardiovascular, gastrointestinal, and orthopedic systems<ref name= ''Cimino''>PMID:29478615</ref>. The wide range of presentations is consistent with the multiplicity of mutations observed in the causative protein<ref name= ''Ly''>PMID:31582003</ref>.





Revision as of 19:33, 7 April 2022

Neurofibromin

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Proteopedia Page Contributors and Editors (what is this?)

Jordyn K. Lenard, Ryan D. Adkins, OCA, Michal Harel, Jaime Prilusky