Discover Community Create You

Loading publication...

Noodle always counts identifier-free feature events for service operation. With your permission, Google Analytics can also estimate audience and engagement. Search text, publication identifiers, cookies, IP addresses and browser fingerprints are never included in Noodle's first-party event counters.

Privacy details

Article

Clinical hematopoietic stem cell-based gene therapy

24 Apr 2025

Abstract excerpt

Hematopoietic stem cell (HSC)-based gene therapies have seen extraordinary progress since their initial conception, now fundamentally transforming the treatment paradigms for various inherited hematologic, immunologic, and metabolic conditions-with additional use cases under exploration. Decades worth of work with advances in viral vector technologies and cell manufacturing have paved the way for HSC gene therapy...

Read the complete abstract on PubMed

Topics

Share this publication in a Topic to start or enrich a Post.

Share to Biomedical Ethics and Regulation
Share to CRISPR and Genetic Engineering
Share to Virus-based gene therapy research

Methodology Data sources Editorial policy Privacy Delete account & data About Contact Helena Bioinformatics

Source

Identifiers

PMID: 40285354
DOI: 10.1016/j.ymthe.2025.04.029

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 23 Oct 2026.

Related research

Semantic proximity does not establish scientific evidence. Graph snapshot: 2026-09-29.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Clinical hematopoietic stem cell-based gene therapyPMID 40285354Read ↗

Therapy Development by Genome Editing of Hematopoietic Stem CellsSemantic proximity Read ↗

The changing landscape of gene editing in hematopoietic stem cells: a step towards Cas9 clinical translationSemantic proximity Read ↗

CRISPR/Cas9-modified hematopoietic stem cells—present and future perspectives for stem cell transplantationSemantic proximity Read ↗

Gene Editing of Human Hematopoietic Stem and Progenitor Cells: Promise and Potential HurdlesSemantic proximity Read ↗

In Vivo Hematopoietic Stem Cell Genome Editing: Perspectives and LimitationsSemantic proximity Read ↗

CRISPR/Cas9 genome editing in human hematopoietic stem cells.Semantic proximity Read ↗

Advancements in Hematopoietic Stem Cell Gene Therapy: A Journey of Progress for Viral TransductionSemantic proximity Read ↗

Highly Efficient Genome Editing of Murine and Human Hematopoietic Progenitor Cells by CRISPR/Cas9Semantic proximity Read ↗

Gene Therapy of HemoglobinopathiesSemantic proximity Read ↗

Homology-Directed-Repair-Based Genome Editing in HSPCs for the Treatment of Inborn Errors of Immunity and Blood DisordersSemantic proximity Read ↗

Viral-based gene therapy clinical trials for immune deficiencies and blood disorders from 2013 until 2023 - an overviewSemantic proximity Read ↗

Understanding genetic heterogeneity in gene-edited hematopoietic stem cell products.Semantic proximity Read ↗

⌁Select a neighboring publication to make it the new centre.