Silencing of B4Galnt1 Gene Prevents GM2 Accumulation in Tay-Sachs Cells

<div>Immunocytochemical analysis of GM2 and Lamp1 in control and TSD human neuroglia. TSD human neuroglia was transduced with <svg height="11.9413pt" id="M110" style="vertical-align:-0.3499002pt" version="1.1" viewbox="-0.0498162 -11.5914 37.3068 11.9413" width="37.3068pt" xmlns="http://www.w3.org/2000/svg" xmlns:xlink="http://www.w3.org/1999/xlink"><g transform="matrix(.013,0,0,-0.013,0,0)"><path d="M137 343C167 482 260 545 321 574C357 591 397 603 429 609L423 641C382 634 335 622 295 608C189 570 37 457 37 238C37 84 125 -12 242 -12C362 -12 447 89 447 209C447 311 374 393 267 393C247 393 226 386 204 376L137 343ZM227 337C318 337 361 256 361 173C361 105 336 22 258 22C176 22 126 120 126 240C126 266 127 291 132 310C155 323 189 337 227 337Z"></path></g><g transform="matrix(.013,0,0,-0.013,9.146,0)"><path d="M528 54L331 254L528 455L492 493L294 291L96 493L60 455L257 254L60 54L96 16L294 217L492 16L528 54Z"></path></g><g transform="matrix(.013,0,0,-0.013,19.682,0)"><path d="M384 0V27C293 34 287 42 287 114V635C232 613 172 594 109 583V559L157 557C201 555 205 550 205 499V114C205 42 199 34 109 27V0H384Z"></path></g><g transform="matrix(.013,0,0,-0.013,25.922,0)"><path d="M241 635C89 635 35 457 35 312C35 153 89 -12 240 -12C390 -12 443 166 443 312C443 466 390 635 241 635ZM238 602C329 602 354 454 354 312C354 172 330 22 240 22C152 22 124 173 124 313S148 602 238 602Z"></path></g><g transform="matrix(.0091,0,0,-0.0091,32.209,-5.741)"><path d="M247 635C116 635 39 518 39 421C39 315 113 237 221 237C239 237 258 241 281 253L349 289C315 140 204 42 60 19L66 -15C90 -15 153 -5 207 16C344 70 447 199 447 385C447 521 373 635 247 635ZM231 598C329 598 356 478 356 390C356 371 355 347 352 325C330 309 297 297 262 297C179 297 127 369 127 456C127 515 156 598 231 598Z"></path></g></svg> viral particles of AAV9-h-shRNA1 expressing mCherry reporter gene (red) in 24-well plates. Representative fluorescent images showed (a–c) GM2 ganglioside and (e–g) lysosome levels in the untreated and AAV9-transduced cells with anti-GM2 (green) and anti-Lamp1 (green) antibodies following 72 hours post-transduction. The total intensity of fluorescence was quantified using Fiji imaging software. 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Advances in Cell and Gene Therapy

fig7

Figure 7

Figure 7: Silencing of B4Galnt1 Gene Prevents GM2 Accumulation in Tay-Sachs Cells 