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Si us plau utilitzeu sempre aquest identificador per citar o enllaçar aquest document: https://hdl.handle.net/2445/213223
Leveraging single-cell genomics for cell therapy in Huntington's disease
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brain regions,particularlyaffectingthestriatalneuronsknownasMediumSpinyNeurons(MSNs).
Unfortunately,neuronsdonotregenerate,thus,makingitimpossibleforthebodytoreplacetheir
function. Despiteextensiveresearch,thereiscurrentlynocureforHD,andthegoaloftreatmentis
to alleviatesymptoms.
Stem celltherapyrepresentsapromisingdirection,allowingindefinite in vitro cells expansionand
differentiationintothedesiredtargetneurons.Themainlimitationofneuralcellstransplantisthat
once theirprojectionsaregrown,theycannotbedetractedfromthecultureplatewithoutbreaking
and causingcelldeath.
In thisproject,wefocusonidentifyingtheoptimaltargetprogenitorsubpopulation.Thecellsshould
be matureenoughtoexclusivelydifferentiateintoMSNsbutyetnotoverlymature,preventingthe
development oftheprojectionsthatleadtocelldeathuponbreaking.Leveragingcomputational
genomics, weanalyzehumansingle-cellRNAsequencingdatatocharacterizetheMSNdifferen-
tiation process.Integrating in vivo and in vitro data, wegroupcellsindifferentclustersdepending
on theirgeneticexpressionandcomputetheirvelocitiestoanalysetheirfate.Ourfindingsreveala
potential groupofcellsthatseemtofulfillallthecriteria.Additionally,theprojecthasenabledthe
characterization ofthedifferentlineagesofcellswithinthedifferentiationprocessofstriatalneurons.
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Treballs Finals de Grau d'Enginyeria Biomèdica. Facultat de Medicina i Ciències de la Salut. Universitat de Barcelona. Curs: 2023-2024. Tutor/Director: Jordi Colomer ; Director: Jordi Abante
Matèries (anglès)
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LOZANO GARGALLO, Clàudia. Leveraging single-cell genomics for cell therapy in Huntington's disease. [consulta: 13 de gener de 2026]. [Disponible a: https://hdl.handle.net/2445/213223]