Back to search

Article

Lentiviral hematopoietic stem and progenitor cell gene therapy provides durable clinical benefit in early-symptomatic early-juvenile metachromatic leukodystrophy

2021-02-01

Abstract excerpt

No abstract is available from the source.

Topics

Open a Topic to create a Post that cites this publication.

Identifiers and source

Literature Corpus work
ff48c53d-72ae-5e27-9118-393ae4dd240a
DOI
10.1016/j.ymgme.2020.12.081
Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest
Interactive article relationship graphSelect a related publication card to move it into the centre and load its closest explainable connections. Solid lines are source-backed structured connections. Dashed lines are semantic discovery signals and are not scientific evidence.
Lentiviral hematopoietic stem and progenitor cell gene therapy provides durable clinical benefit in early-symptomatic early-juvenile metachromatic leukodystrophyDOI 10.1016/j.ymgme.2020.12.081
Lentivirus-modified hematopoietic stem cell gene therapy for advanced symptomatic juvenile metachromatic leukodystrophy: a long-term follow-up pilot studySemantic proximity
Gene therapy targeting haematopoietic stem cells for inherited diseases: progress and challengesSemantic proximity
In vivo gene therapy of metachromatic leukodystrophy by lentiviral vectors: correction of neuropathology and protection against learning impairments in affected miceSemantic proximity
Exploring the genetic and epigenetic origins of juvenile myelomonocytic leukemia using newborn screening samples.Semantic proximity
Outcome of Early Juvenile Onset Metachromatic Leukodystrophy After Unrelated Cord Blood Transplantation: A Case Series and Review of the LiteratureSemantic proximity
Hematopoietic Stem Cell Transplantation to Treat Leukodystrophies: Clinical Practice Guidelines from the Hunter's Hope Leukodystrophy Care NetworkSemantic proximity
Emerging treatments for pediatric leukodystrophies.Semantic proximity
New Indications for Hematopoietic Stem Cell Gene Therapy in Lysosomal Storage DisordersSemantic proximity
Enzyme, cell and gene‐based therapies for metachromatic leukodystrophySemantic proximity
Long-term outcome of haematopoietic stem cell transplantation in autosomal recessive osteopetrosis: an EBMT reportSemantic proximity
A Mutation-Agnostic Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy.Semantic proximity
Gene therapy for metachromatic leukodystrophySemantic proximity
Select a neighboring publication to make it the new centre.