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| http://dx.doi.org/10.1007/s43465-022-00809-y | DOI Listing |
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Refractory myasthenia gravis treated with autologous hematopoietic stem cell transplantation.
Ann Clin Transl Neurol
December 2024
Transplant and Cell Therapy Program, Division of Hematology, Department of Medicine, The Ottawa Hospital, Ottawa, Ontario, Canada.
Objectives: Patients with refractory myasthenia gravis (MG) have few treatment options. Autologous hematopoietic stem cell transplantation (HSCT) has been used to treat immune diseases; however, its use in the treatment of MG is not broadly considered. Our objective is to report on the efficacy and safety of HSCT in refractory MG.
View Article and Find Full Text PDFSimoultaneous bilateral medial opening wedge high tibial osteotomy can be performed safely and effectively without bone grafting: analysis of a monocentric retrospective series.
BMC Musculoskelet Disord
December 2024
IRCCS Ospedale Sacro Cuore-Don Calabria; Viale Luigi Rizzardi 4, Negrar, VR, Italia.
Background: Simultaneous bilateral high tibial osteotomy (SBHTO) is a potential solution to treat bilateral medial osteoarthritis (OA) associated with tibial varus deformity. Concerns on the potential problems related to bone healing exists, and most of the surgeon performing SBHTO use bone void filler as associated procedure. This paper aim to evaluate safety and efficacy of SBHTO using locking plate, autologous cancellous bone mobilization and no bone void filler with immediate weight bearing at tolerance protocol.
View Article and Find Full Text PDFAutologous DNA mobilization and multiplication expedite natural products discovery from bacteria.
Science
December 2024
Helmholtz Institute for Pharmaceutical Research Saarland (HIPS), Helmholtz Centre for Infection Research (HZI), Saarbrücken, Germany.
The transmission of antibiotic-resistance genes, comprising mobilization and relocation events, orchestrates the dissemination of antimicrobial resistance. Inspired by this evolutionarily successful paradigm, we developed ACTIMOT, a CRISPR-Cas9-based approach to unlock the vast chemical diversity concealed within bacterial genomes. ACTIMOT enables the efficient mobilization and relocation of large DNA fragments from the chromosome to replicative plasmids within the same bacterial cell.
View Article and Find Full Text PDFHaematopoietic gene therapy of non-conditioned patients with Fanconi anaemia-A: results from open-label phase 1/2 (FANCOLEN-1) and long-term clinical trials.
Lancet
December 2025
Biomedical Innovation Unit, Center for Research on Energy, Environment and Technology (CIEMAT), Madrid, Spain; Biomedical Network Research Center for Rare Diseases (CIBERER), Madrid, Spain; Sanitary Research Institute Fundación Jiménez Díaz (U.A.M), Madrid, Spain. Electronic address:
Background: Allogeneic haematopoietic stem-cell transplantation is the standard treatment for bone marrow failure (BMF) in patients with Fanconi anaemia, but transplantation-associated complications such as an increased incidence of subsequent cancer are frequent. The aim of this study was to evaluate the safety and efficacy of the infusion of autologous gene-corrected haematopoietic stem cells as an alternative therapy for these patients.
Methods: This was an open-label, investigator-initiated phase 1/2 clinical trial (FANCOLEN-1) and long-term follow-up trial (up to 7 years post-treatment) in Spain.
Pre-emptive increase in the filgrastim dose based on the CD34 cell count on day four of autologous stem cell mobilization.
Hematol Transfus Cell Ther
December 2024
Universidade Federal de Juiz de Fora (UFJF), Juiz de Fora, MG, Brazil.
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