Caveat to protein replacement therapy for genetic disease. Immunological implications of accurate molecular diagnosis

Late onset of severe graft-versus-host disease in a pediatric liver transplant recipient.

Transplantation·2001

X microchromosome with additional chromosome anomalies found in Ullrich-Turner syndrome.

American journal of medical genetics·1995

Cystic fibrosis-related diabetes is associated with HLA DQB1 alleles encoding Asp-57- molecules.

Journal of clinical immunology·1994

Specific inhibition of formation of transcription complexes by a calicheamicin oligosaccharide: a paradigm for the development of transcriptional antagonists.

Proceedings of the National Academy of Sciences of the United States of America·1994

Roles of erythropoietin, insulin-like growth factor 1, and unidentified serum factors in promoting maturation of purified murine erythroid colony-forming units.

Blood·1992

Onset of erythropoietin response in murine erythroid colony-forming units: assignment to early S-phase in a specific cell generation.

Blood·1992

Efficacy and safety of garetosmab, an activin A-blocking antibody, in fibrodysplasia ossificans progressiva (OPTIMA): a randomised, double-blind, placebo-controlled, phase 3 trial.

Lancet (London, England)·2026

Radiotherapy versus observation following surgical resection of WHO grade 2 atypical meningioma (ROAM/EORTC-1308): an international, multicentre, open-label, phase 3, randomised controlled trial.

Lancet (London, England)·2026

Faecal microbiota transplantation in irritable bowel syndrome (REFIT2): a randomised, double-blind, placebo-controlled, phase 3 trial.

Lancet (London, England)·2026

Understanding peripheral arterial disease in the current era.

Lancet (London, England)·2026

Current advancements in the care of peripheral arterial disease: modern approaches to medical, endovascular, and surgical treatment.

Lancet (London, England)·2026

[Anti-VEGF treatment in age related macular degeneration].

La Revue du praticien·2026

Targeting O6-methylguanine-DNA Methyltransferase Deficiency in Preclinical Models of Extracranial Human Cancers with a Tumor-Selective DNA-Modifying Agent.

Molecular cancer therapeutics·2026

"The pharmacological potential of CRISPR-Cas9: Revolutionizing gene therapy and drug development".

Nucleosides, nucleotides & nucleic acids·2026

Cardiac Imaging in Structural Valve Clinical Trials: Secondary Mitral Regurgitation.

Circulation. Cardiovascular imaging·2026

Corrigendum to 'A triple-mode strategy on JQ1-loaded nanoplatform for superior antitumor therapy in pancreatic cancer' [Mater Today Bio. 2025 Mar 21;32:101696].

Materials today. Bio·2026

Case Report: Hereditary transthyretin amyloidosis in a Chinese family with the transthyretin variant p.Y89H.

Frontiers in neurology·2026

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Caveat to protein replacement therapy for genetic disease. Immunological implications of accurate molecular diagnosis

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Late onset of severe graft-versus-host disease in a pediatric liver transplant recipient.

X microchromosome with additional chromosome anomalies found in Ullrich-Turner syndrome.

Cystic fibrosis-related diabetes is associated with HLA DQB1 alleles encoding Asp-57- molecules.

Specific inhibition of formation of transcription complexes by a calicheamicin oligosaccharide: a paradigm for the development of transcriptional antagonists.

Roles of erythropoietin, insulin-like growth factor 1, and unidentified serum factors in promoting maturation of purified murine erythroid colony-forming units.

Onset of erythropoietin response in murine erythroid colony-forming units: assignment to early S-phase in a specific cell generation.

Efficacy and safety of garetosmab, an activin A-blocking antibody, in fibrodysplasia ossificans progressiva (OPTIMA): a randomised, double-blind, placebo-controlled, phase 3 trial.

Radiotherapy versus observation following surgical resection of WHO grade 2 atypical meningioma (ROAM/EORTC-1308): an international, multicentre, open-label, phase 3, randomised controlled trial.

Faecal microbiota transplantation in irritable bowel syndrome (REFIT2): a randomised, double-blind, placebo-controlled, phase 3 trial.

Understanding peripheral arterial disease in the current era.

Current advancements in the care of peripheral arterial disease: modern approaches to medical, endovascular, and surgical treatment.

[Anti-VEGF treatment in age related macular degeneration].

Targeting O6-methylguanine-DNA Methyltransferase Deficiency in Preclinical Models of Extracranial Human Cancers with a Tumor-Selective DNA-Modifying Agent.

"The pharmacological potential of CRISPR-Cas9: Revolutionizing gene therapy and drug development".

Cardiac Imaging in Structural Valve Clinical Trials: Secondary Mitral Regurgitation.

Corrigendum to 'A triple-mode strategy on JQ1-loaded nanoplatform for superior antitumor therapy in pancreatic cancer' [Mater Today Bio. 2025 Mar 21;32:101696].

Case Report: Hereditary transthyretin amyloidosis in a Chinese family with the transthyretin variant p.Y89H.

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Caveat to protein replacement therapy for genetic disease. Immunological implications of accurate molecular diagnosis

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