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	201.	Two breakthrough gene-targeted treatments for spinal muscular atrophy: challenges remain. [electronic resource] by Sumner, Charlotte J Crawford, Thomas O Producer: 20190910 In: The Journal of clinical investigation vol. 128 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	202.	A novel role of U1 snRNP: Splice site selection from a distance. [electronic resource] by Singh, Ravindra N Singh, Natalia N Producer: 20191015 In: Biochimica et biophysica acta. Gene regulatory mechanisms vol. 1862 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	203.	[A pilot study on spinal muscular atrophy carrier screening in Shanghai region using real-time PCR]. [electronic resource] by Qu, Xiao-xing Xiao, Bing Ji, Xing Jiang, Wen-ting Yang, Zu-jing Tao, Jiong Producer: 20130726 In: Zhonghua yi xue yi chuan xue za zhi = Zhonghua yixue yichuanxue zazhi = Chinese journal of medical genetics vol. 30 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	204.	Translational fidelity of intrathecal delivery of self-complementary AAV9-survival motor neuron 1 for spinal muscular atrophy. [electronic resource] by Passini, Marco A Bu, Jie Richards, Amy M Treleaven, Christopher M Sullivan, Jennifer A O'Riordan, Catherine R Scaria, Abraham Kells, Adrian P Samaranch, Lluis San Sebastian, Waldy Federici, Thais Fiandaca, Massimo S Boulis, Nicholas M Bankiewicz, Krystof S Shihabuddin, Lamya S Cheng, Seng H Producer: 20150313 In: Human gene therapy vol. 25 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	205.	Molecular evolution of the moonlighting protein SMN in metazoans. [electronic resource] by Stump, Aram D Dillon-White, Marsha Gu, Shaun Producer: 20140314 In: Comparative biochemistry and physiology. Part D, Genomics & proteomics vol. 8 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	206.	Enhanced Carrier Screening for Spinal Muscular Atrophy: Detection of Silent (SMN1: 2 + 0) Carriers Utilizing a Novel TaqMan Genotyping Method. [electronic resource] by Azad, Abul Kalam Huang, Chih-Kang Jin, Hong Zou, Hongwei Yanakakis, Lindsay Du, Juan Fiddler, Morry Naeem, Rizwan Goldstein, Yitz Producer: 20210420 In: Laboratory medicine vol. 51 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	207.	Effect of diet on the survival and phenotype of a mouse model for spinal muscular atrophy. [electronic resource] by Butchbach, Matthew E R Rose, Ferrill F Rhoades, Sarah Marston, John McCrone, John T Sinnott, Rachel Lorson, Christian L Producer: 20100315 In: Biochemical and biophysical research communications vol. 391 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	208.	Homozygous deletion in the SMN1 gene in asymptomatic individual - genetic counselling issues in SMA-risk families. [electronic resource] by Jędrzejowska, Maria Szczałuba, Krzysztof Sielska, Danuta Producer: 20120109 In: Medycyna wieku rozwojowego vol. 15 Availability: No items available. Save to lists Add to cart (remove)
	209.	Clinical and molecular characterization of patients with gross hypotonia and impaired lower motor neuron function. [electronic resource] by Sheth, Jayesh Patel, Harsh Mehta, Sanjiv Tewari, Stuti Sheth, Frenny Producer: 20140317 In: Indian pediatrics vol. 50 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	210.	SMA prenatal diagnosis: a modified protocol to help differentiation between deletions and gene conversion. [electronic resource] by Kekou, K Sofocleous, C Konstantinidis, G Fryssira, H Mavrou, A Kitsiou, S Kanavakis, E Producer: 20151110 In: Molecular and cellular probes vol. 29 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	211.	Synthetic Nucleic Acids and Treatment of Neurological Diseases. [electronic resource] by Corey, David R Producer: 20170529 In: JAMA neurology vol. 73 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	212.	Pilot study of population-based newborn screening for spinal muscular atrophy in New York state. [electronic resource] by Kraszewski, Jennifer N Kay, Denise M Stevens, Colleen F Koval, Carrie Haser, Bianca Ortiz, Veronica Albertorio, Anthony Cohen, Lilian L Jain, Ritu Andrew, Sarah P Young, Sally Dunaway LaMarca, Nicole M De Vivo, Darryl C Caggana, Michele Chung, Wendy K Producer: 20181017 In: Genetics in medicine : official journal of the American College of Medical Genetics vol. 20 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	213.	Splice-site pairing is an intrinsically high fidelity process. [electronic resource] by Fox-Walsh, Kristi L Hertel, Klemens J Producer: 20090310 In: Proceedings of the National Academy of Sciences of the United States of America vol. 106 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	214.	Restoration of full-length SMN promoted by adenoviral vectors expressing RNA antisense oligonucleotides embedded in U7 snRNAs. [electronic resource] by Geib, Till Hertel, Klemens J Producer: 20100317 In: PloS one vol. 4 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	215.	Genetic therapy for spinal muscular atrophy. [electronic resource] by MacKenzie, Alex Producer: 20100601 In: Nature biotechnology vol. 28 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	216.	Decreased microRNA levels lead to deleterious increases in neuronal M2 muscarinic receptors in Spinal Muscular Atrophy models. [electronic resource] by O'Hern, Patrick J do Carmo G Gonçalves, Inês Brecht, Johanna López Soto, Eduardo Javier Simon, Jonah Chapkis, Natalie Lipscombe, Diane Kye, Min Jeong Hart, Anne C Producer: 20180209 In: eLife vol. 6 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	217.	Moving towards treatments for spinal muscular atrophy: hopes and limits. [electronic resource] by Wirth, Brunhilde Barkats, Martine Martinat, Cecile Sendtner, Michael Gillingwater, Thomas H Producer: 20160404 In: Expert opinion on emerging drugs vol. 20 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	218.	α-COP binding to the survival motor neuron protein SMN is required for neuronal process outgrowth. [electronic resource] by Li, Hongxia Custer, Sara K Gilson, Timra Hao, Le Thi Beattie, Christine E Androphy, Elliot J Producer: 20160920 In: Human molecular genetics vol. 24 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	219.	Critical period of neuromuscular development: Importance for a new treatment of SMA. [electronic resource] by Vrbová, Gerta Sławińska, Urszula Producer: 20191024 In: Neuromuscular disorders : NMD vol. 28 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	220.	The importance of the SMN genes in the genetics of sporadic ALS. [electronic resource] by Corcia, Philippe Camu, William Praline, Julien Gordon, Paul H Vourch, Patrick Andres, Christian Producer: 20100218 In: Amyotrophic lateral sclerosis : official publication of the World Federation of Neurology Research Group on Motor Neuron Diseases vol. 10 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)

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