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Cell therapies for inherited myopathies

2003· review· en· W2077924804 on OpenAlexaff
Daniel Skuk, Jacques P. Tremblay

Bibliographic record

VenueCurrent Opinion in Rheumatology · 2003
Typereview
Languageen
FieldBiochemistry, Genetics and Molecular Biology
TopicMuscle Physiology and Disorders
Canadian institutionsCentre hospitalier de l'Université Laval
Fundersnot available
KeywordsMyocyteMedicineCell therapyTransplantationPopulationImmunologyContext (archaeology)CellStem cellCell typeCell biologyCancer researchBiologyInternal medicineGenetics

Abstract

fetched live from OpenAlex

PURPOSE OF REVIEW: Cell therapies for inherited myopathies are based on the implantation of normal or genetically corrected myogenic cells into the body. This review summarizes the recent progress in this field, systematized according to the factors important for success. RECENT FINDINGS: In the choice of donor cells, myoblasts derived from satellite cells remain the best choice. Some studies on the population of muscle-derived stem cells in mice suggested that these cells may have some advantages over myoblasts; however, no results supporting this advantage have been presented in a primate model. Recent studies on bone marrow transplantation as a systemic source of myogenic precursors for the treatment of myopathies were disappointing. Concerning donor cell delivery, intramuscular myoblast injection remains the only way that can significantly introduce exogenous myogenic cells into the muscles. A recent study in primates showed some parameters of myoblast injection that could be useful in the human. Progress was made in mice to understand the factors that could favor the migration of the donor myoblasts in the host muscles. Concerning donor cell survival, analysis of immune cell infiltration dynamics allowed a better understanding of the factors implicated in early donor cell death. Progress was made on the control of acute rejection for myoblast transplantation in primates. So far, few mouse experiments have advanced the field of tolerance induction toward myogenic cells. SUMMARY: Myoblast transplantation (intramuscular injection of satellite cell-derived myoblasts) currently remains the only cell-based therapy that has produced promising results in the context of a preclinical model such as the nonhuman primate.

Fetched live from OpenAlex and de-inverted. Abstracts are not stored in this database: the inverted indexes are 8.6 GB of the frame’s 9.3 GB of text, and the host has 13 GB free.

How this classification was reachedexpand

Full frame distilled prediction

Teacher imitation

Not calibrated prevalence, not ground truth. Human validation pending. Learned from the 10,348 direct Codex labels and 10,348 direct Gemma labels. Candidate is the union of thresholded teacher heads; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels or direct frontier model labels.

metaresearch head score (Codex)0.000
metaresearch head score (Gemma)0.000
Version: codex-gemma-dda1882f352aValidation status: machine_predicted_unvalidated
Candidate categoriesMeta-epidemiology (narrow)
Consensus categoriesnone
DomainCandidate signal: none · Consensus signal: none
Study designCandidate signal: Not applicable · Consensus signal: none
GenreCandidate signal: Review · Consensus signal: Review
Teacher disagreement score0.904
Threshold uncertainty score1.000

Codex and Gemma teacher scores by category

CategoryCodexGemma
Metaresearch0.0000.000
Meta-epidemiology (narrow)0.0000.000
Meta-epidemiology (broad)0.0010.000
Bibliometrics0.0000.000
Science and technology studies0.0000.000
Scholarly communication0.0000.000
Open science0.0000.000
Research integrity0.0010.000
Insufficient payload (model declined to judge)0.0000.000

Machine scores (provisional)

The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.

Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.

Opus teacher head0.075
GPT teacher head0.382
Teacher spread0.307 · how far apart the two teachers sit on this one work
Validation statusscore_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from it

Classification

machine, unvalidated

Machine predicted; a candidate call from one teacher head, not a consensus.

Study designNot applicable
Domainnot available
GenreReview

How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".

Quick stats

Citations23
Published2003
Admission routes1
Has abstractyes

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