Main research areas: Development of gene editing tools; Development of efficient delivery methods; Development of gene therapy for monogenic diseases, such as sickle cell anemia and epidermolysis bullosa.

Technological skills of the group: Design of editing tools (CRISPR, Base Editing, Prime Editing, Recombinases) - Cloning by multiple strategies - In vitro transcription (mRNAs) - PCR, qPCR, dPCR - Sanger sequencing and NGS (with bioinformatics analysis) - Functional assays for hematopoietic stem cells in vitro (includes Erythroid Diffusion; Methocult, CFA). - Multiparametric cytometry and panels focused on the biology and functionality of stem cells (and immune cells) - HPLC (especially globin cytology) - Delivery methods (nucleoporation, viruses, LNPs, liposomes, and VLPs) - In vivo functional assays (NBSGW mice) - Creation of 3D skin from fibroblasts and keratinocytes.

Key laboratory equipment: Nucleoporators (Lonza and Maxcyte) Sequencers (Sanger and NGS) qPCR and dPCR Cytometers (reading, sorting/single-cell sorting, spectral) NB2 cleanroom infrastructure for gene therapy In vivo experimentation infrastructure (immunocompromised mice).

Social media: @geneall.ciência (Instagram, LinkedIn, TikTok) and www.geneall.org