Results 61 to 70 of about 7,531,202 (384)
GENE THERAPY IN MUCOPOLYSACCHARIDOSIS TYPE IIIA: CASE REPORTS
Slovenska pediatrija, 2022 Mucopolysaccharidoses are a group of rare lysosomal storage diseases. The clinical signs develop gradually, the impairment is progressive and multiple organs are affected. With the currently known treatment options, the patient cannot be cured.
Benjamin Lah +7 more
doaj +1 more source
Diphthamide synthesis is linked to the eEF2‐client chaperone machinery
FEBS Letters, EarlyView.The diphthamide modification of eukaryotic translation elongation factor 2 (eEF2) is important for accurate protein synthesis. We addressed the potential coordination of de novo eEF2 synthesis with simultaneous or subsequent diphthamide modification. Our work reveals that the co‐chaperones Hgh1 and Cpr7, which are known to support folding of nascent ...
Lars Kaduhr +4 more
wiley +1 more source
Genetic treatment of a molecular disorder: gene therapy approaches to sickle cell disease.
Blood, 2016 Effective medical management for sickle cell disease (SCD) remains elusive. As a prevalent and severe monogenic disorder, SCD has been long considered a logical candidate for gene therapy.
Megan D Hoban, S. Orkin, D. E. Bauer
semanticscholar +1 more source
FEBS Letters, EarlyView.Low‐density lipoprotein receptor‐related protein 6 (LRP6) is a key receptor for the Wnt antagonist Dickkopf1 (DKK1). DKK1 protein expression is induced in a bleomycin (BLM)‐induced lung injury model. We show that DKK1 induces proinflammatory and profibrotic genes in lung fibroblasts.
Eun‐Ah Sung +6 more
wiley +1 more source
A mathematical model of Breast cancer (ER+) with excess estrogen: Mixed treatments using Ketogenic diet, endocrine therapy and Immunotherapy [PDF]
arXiv, 2022 Breast Cancer is a major public health problem and the most common diagnosed malignancy in woman. There have been significant developments in clinical approaches and theoretical experimental to understand the interactions of cancer cells dynamics with the immune system, also developments on analytical and computational models to help provide insights ...
arxiv
Role of gene therapy in treatment of cancer for craniofacial regeneration—current molecular strategies, future perspectives, and challenges: a narrative review [PDF]
Journal of Yeungnam Medical ScienceGene therapy involves the introduction of foreign genetic material into host tissue to alter the expression of genetic products. Gene therapy represents an opportunity to alter the course of various diseases.
Himanshu Singh
doaj +1 more source
Journal of Hematology & Oncology, 2017 BackgroundThe majority of individuals with acute myeloid leukemia (AML) respond to initial chemotherapy and achieve a complete remission, yet only a minority experience long-term survival because a large proportion of patients eventually relapse with ...
H. Hackl, K. Astanina, R. Wieser
semanticscholar +1 more source
Natural killer cells in cancer biology and therapy
Molecular Cancer, 2020 The tumor microenvironment is highly complex, and immune escape is currently considered an important hallmark of cancer, largely contributing to tumor progression and metastasis.
Songyang Wu +3 more
semanticscholar +1 more source
Nephrotic Syndrome: Genetics, Mechanism, and Therapies [PDF]
BioMed Research International, 2018 ispartof: location:United ...
Jae Il Shin +3 more
openaire +5 more sources
Characteristics of the Kelch domain containing (KLHDC) subfamily and relationships with diseases
FEBS Letters, EarlyView.The Kelch protein superfamily includes 63 members, with the KLHDC subfamily having 10 proteins. While their functions are not fully understood, recent advances in KLHDC2's structure and role in protein degradation have highlighted its potential for drug development, especially in PROTAC therapies.
Courtney Pilcher +6 more
wiley +1 more source