Shilpita Sarcar

34-38 Provost Street
London, ENG N17NG, United Kingdom

Last Updated: 09/26/2026

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Found 8 publications

KCNV2-Deficient Retinal Organoid Model of Cone Dystrophy-In Vitro Screening for AAV Gene Replacement Therapy.

KCNV2-Deficient Retinal Organoid Model of Cone Dystrophy-In Vitro Screening for AAV Gene Replacement Therapy.

Journal: International journal of molecular sciences

Published: November 13, 2025

3D human induced pluripotent stem cell-derived bioengineered skeletal muscles for tissue, disease and therapy modeling.

3D human induced pluripotent stem cell-derived bioengineered skeletal muscles for tissue, disease and therapy modeling.

Journal: Nature protocols

Published: November 01, 2021

Modeling Skeletal Muscle Laminopathies Using Human Induced Pluripotent Stem Cells Carrying Pathogenic LMNA Mutations.

Modeling Skeletal Muscle Laminopathies Using Human Induced Pluripotent Stem Cells Carrying Pathogenic LMNA Mutations.

Journal: Frontiers in physiology

Published: March 15, 2018

Three-Dimensional Human iPSC-Derived Artificial Skeletal Muscles Model Muscular Dystrophies and Enable Multilineage Tissue Engineering.

Three-Dimensional Human iPSC-Derived Artificial Skeletal Muscles Model Muscular Dystrophies and Enable Multilineage Tissue Engineering.

Journal: Cell reports

Published: November 15, 2016

Efficient In Vivo Liver-Directed Gene Editing Using CRISPR/Cas9.

Efficient In Vivo Liver-Directed Gene Editing Using CRISPR/Cas9.

Journal: Molecular therapy : the journal of the American Society of Gene Therapy

Published: December 21, 2015

Next-generation muscle-directed gene therapy by in silico vector design.

Next-generation muscle-directed gene therapy by in silico vector design.

Journal: Nature communications

Published: November 15, 2015

Genome-wide computational analysis reveals cardiomyocyte-specific transcriptional Cis-regulatory motifs that enable efficient cardiac gene therapy.

Genome-wide computational analysis reveals cardiomyocyte-specific transcriptional Cis-regulatory motifs that enable efficient cardiac gene therapy.

Journal: Molecular therapy : the journal of the American Society of Gene Therapy

Published: April 16, 2014

Computationally designed liver-specific transcriptional modules and hyperactive factor IX improve hepatic gene therapy.

Computationally designed liver-specific transcriptional modules and hyperactive factor IX improve hepatic gene therapy.

Journal: Blood

Published: March 19, 2014

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Last Updated: 09/26/2026