Zhou, Miaojin <br/><br/>
Seamless Genetic Conversion of SMN2 to SMN1 via CRISPR/Cpf1 and Single-Stranded Oligodeoxynucleotides in Spinal Muscular Atrophy Patient-Specific Induced Pluripotent Stem Cells.  [electronic resource] 

 -  Human gene therapy  Nov 2018 
 - 1252-1263 p.  digital 
<br/><br/> Publication Type: Journal Article; Research Support, Non-U.S. Gov't 
<br/><br/><label>ISSN: </label>1557-7422 
<br/><br/><label>Standard No.: </label>10.1089/hum.2017.255  doi 
<br/><br/><label>Subjects--Topical Terms: </label><br/>Base Sequence<br/>Cell Differentiation<br/>Clone Cells<br/>Clustered Regularly Interspaced Short Palindromic Repeats--genetics<br/>Gene Conversion<br/>Genotype<br/>Humans<br/>Induced Pluripotent Stem Cells--metabolism<br/>Male<br/>Motor Neurons--metabolism<br/>Muscular Atrophy, Spinal--genetics<br/>Oligodeoxyribonucleotides--metabolism<br/>Proto-Oncogene Proteins c-myc--metabolism<br/>RNA, Guide, CRISPR-Cas Systems--metabolism<br/>Survival of Motor Neuron 1 Protein--genetics<br/>Survival of Motor Neuron 2 Protein--genetics<br/>Young Adult