A scalable and cGMP-compatible autologous organotypic cell therapy for Dystrophic Epidermolysis Bullosa.

doi:10.1038/s41467-024-49400-z

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Article

A scalable and cGMP-compatible autologous organotypic cell therapy for Dystrophic Epidermolysis Bullosa.

Nature communications - 11 Jul 2024

Neumayer Gernot, Torkelson Jessica L, Li Shengdi, McCarthy Kelly, Zhen Hanson H, Vangipuram Madhuri, Mader Marius M, Gebeyehu Gulilat, Jaouni Taysir M, Jacków-Malinowska Joanna, Rami Avina, Hansen Corey, Guo Zongyou, Gaddam Sadhana, Tate Keri M, Pappalardo Alberto, Li Lingjie, Chow Grace M, Roy Kevin R, Nguyen Thuylinh Michelle, Tanabe Koji, McGrath Patrick S, Cramer Amber, Bruckner Anna, Bilousova Ganna, Roop Dennis, Tang Jean Y, Christiano Angela, Steinmetz Lars M, Wernig Marius, Oro Anthony E

Abstract excerpt

We present Dystrophic Epidermolysis Bullosa Cell Therapy (DEBCT), a scalable platform producing autologous organotypic iPS cell-derived induced skin composite (iSC) grafts for definitive treatment. Clinical-grade manufacturing integrates CRISPR-mediated genetic correction with reprogramming into one step, accelerating derivation of COL7A1-edited iPS cells from patients. Differentiation into epidermal, dermal and...

Topics

Humans
Epidermolysis Bullosa Dystrophica
Animals
Induced Pluripotent Stem Cells
Mice
Collagen Type VII
Cell- and Tissue-Based Therapy

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Fibroblasts

Cell Differentiation

Keratinocytes

Skin

Transplantation, Autologous

Male

Mutation

Female

Skin Transplantation

Gene Editing

CRISPR-Cas Systems

Source

Open in PubMed Open full text

Identifiers

PMID: 38992003
DOI: 10.1038/s41467-024-49400-z
PMCID: PMC11239819

Genes

COL7A1

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 31 Aug 2026.

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