Cell Therapy eBook Series
Exploring Electroporation 2: Genetic Diseases
Unlocking the Code: Insights into Genetic Disease Therapy
Genetic engineering has revolutionized our ability to understand, prevent and treat disease. Genomic anomalies cause a myriad of illnesses that range in complexity from monogenetic diseases or multifactorial disorders to chromosomal abnormalities. With the growing arsenal of genome editing tools, precise manipulation of the genome is now possible, giving rise to gene therapies that have the potential to cure genetic disease. Explore how MaxCyte electroporation efficiently and reproducibly delivers CRISPR, RNPs and RNAs for targeted gene correction with the flexibility to support today’s research and satisfy the stringent demands of tomorrow’s clinical use.
Content:
- Ex Vivo Gene Correction in X-CGD Patient Stem Cells
- Hematopoietic Stem Cell Electroporation for HDR-Mediated Correction of an X-Linked Blood Disorder
- Scalable Electroporation of Adult Keratinocytes with Multiplexed CRISPR for Development of Novel Cell Therapeutics
- Large-Scale Engineering of Extracellular Nanoparticles with CRISPR-Cas9 Ribonucleoproteins
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