Tippi C. MacKenzie

Combinatorial base editing couples disease correction with lineage amplification in hematopoietic stem and progenitor cells

This paper develops a combinatorial base editing strategy to enhance erythroid lineage amplification and disease correction in hematopoietic stem and progenitor cells.

Integrated single-nuclei and spatial transcriptomic profiling of human sacrococcygeal teratomas reveals heterogeneity in cellular composition and X-chromosome inactivation

This study uses single-nuclei RNA-seq and spatial transcriptomics to analyze human sacrococcygeal teratomas, revealing cellular heterogeneity and X-chromosome inactivation patterns linked to sex bias.

In utero and neonatal lipid nanoparticle delivery achieves robust editing in hematopoietic stem cells.

This study demonstrates efficient in utero and neonatal lipid nanoparticle delivery for genome editing of hematopoietic stem cells, achieving robust editing and multilineage reconstitution.

In Utero Hematopoietic Stem Cell Transplant for Fanconi Anemia

This study demonstrates that in utero hematopoietic stem cell transplantation (HSCT) without conditioning can effectively engraft donor cells in FA mouse models, achieving high donor chimerism and potentially offering a safe prenatal treatment for FA.

A Multiomics, Spatiotemporal, and Single Cell Atlas for Mapping Cell-Type-Specific Dysregulation at the Maternal-Fetal Interface

This study creates a comprehensive single-cell atlas of the maternal-fetal interface, identifying cell-type-specific dysregulation in pregnancy complications.

Dual α-globin and truncated erythropoietin receptor knock-in restores hemoglobin production in α-thalassemia major-derived hematopoietic stem and progenitor cells

This paper describes a genome editing strategy that restores hemoglobin production in α-thalassemia major-derived hematopoietic stem and progenitor cells by integrating a functional α-globin gene and a truncated erythropoietin receptor.

Systematic evaluation of genome sequencing for the assessment of fetal structural anomalies

This study evaluates genome sequencing as a single diagnostic approach for fetal structural anomalies, showing it detects nearly all variants from standard tests and provides higher diagnostic yield than individual tests.

Large differences in small RNA composition between human biofluids

The paper compares small RNA composition across 12 human biofluids, finding large variations in miRNA and tDR levels and identifying Y RNA fragments in several biofluids.