All individuals with spinal muscular atrophy have at least one “backup gene,” known asSMN2.TheSMN2gene has a similar structure toSMN1, but only a small amount (10%) of the SMN protein it produces is fully functional. This low level of SMN protein is not effective enough to sustain the...
It works by making the SMN2 gene, which acts as a backup, look more like the SMN1 gene and creates the required protein. A spinal tap is used to administer this with four doses given over two months, followed by four doses every four months after that. Studies show that breathing, ...
SMN1 deletionSMN2 copynumberskin necrosisspinal muscular atrophyvasculopathyNeurodegenerative diseases represent a growing public health challenge. Current medications treat symptoms, but none halt or retard neurodegeneration. The recent advent of pluripotent cell biology has opened new avenues for ...
A mutation in theSMN1gene is responsible for the majority of instances of spinal muscular atrophy (SMA). A protein necessary for proper motor neuron activity is not produced in sufficient amounts by this gene. Motor neurons perish and are, thus, unable to transmit muscular impulses. A child w...
SMMMS SMMN SMMNRA SMMO SMMoA SMMOG SMMP SMMPA SMMR SMMRI SMMRP SMMS SMMSD SMMSE SMMSE-IC SMMSO SMMSP SMMT SMMTT SMMU SMMUG SMMUSD SMMV SMMW SMMWR SMMWW SMN SMN1 SMN2 SMNA SMNC SMND SMNDA SMNDC SMNDR SMNF SMNG-A ▼
Pigou and Coase reconsidered On 31 December 2016,SMNP's share price reached 712 bzs per share. Steady performance More results ► Acronyms browser? ▲ SMMU SMMUG SMMUSD SMMV SMMW SMMWR SMMWW SMN SMN1 SMN2 SMNA SMNC SMND SMNDA
SMN SMN1 SMN2 SMNA SMNC SMND SMNDA SMNDC SMNDR SMNF SMNG-A SMNH SMNHA SMNHS SMNHST SMNI SMNN SMNO SMNP SMNR SMNRA SMNRP SMNT SMNTY SMNUG SMNV SMNYL SMNZ SMO SMoA SMOAD SMOAF SMOB SMOBC SMOBE SMOBSMOD SMOC SMOCA ...
SMN1 SMN2 SMNA SMNC SMND SMNDA SMNDC SMNDR SMNF SMNG-A SMNH SMNHA SMNHS SMNHST SMNI SMNN SMNO SMNP SMNR SMNRA SMNRP SMNT SMNTY SMNUG SMNV SMNYL SMNZ SMO SMoA SMOAD SMOAF SMOB SMOBC SMOBE SMOBSMOD SMOC SMOCA SMOCC ...
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A good example of targeting underlying splicing mechanisms to treat diseases is with a disease calledspinal muscular atrophy, a major genetic disease of children and infants. Humans carry two near identical copies of the Survival Motor Neuron gene: SMN1 and SMN2 which are essential for the surviva...