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   Articles with "genetic therapies" as a keyword



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Genetic therapies for cystic fibrosis lung disease

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recommendations! Published in 2017 at "Current Opinion in Pharmacology"

DOI: 10.1016/j.coph.2017.10.006

Abstract: HighlightsGene editing strategies for gene therapy of cystic fibrosis.Chemically modified mRNA for CFTR protein replacement therapy.Better viral and non‐viral vectors targeted to lung epithelium.Human and animal models for CF. &NA; Gene therapy for cystic fibrosis… read more here.

Keywords: non viral; cystic fibrosis; fibrosis; gene therapy ... See more keywords

Genetic Therapies for Hearing Loss: Accomplishments and Remaining Challenges

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recommendations! Published in 2019 at "Neuroscience Letters"

DOI: 10.1016/j.neulet.2019.134527

Abstract: More than 15 years have passed since the official completion of the Human Genome Project. Predominantly due to this project, over one hundred genes have now been linked to hearing loss. Although major advancements have… read more here.

Keywords: hearing loss; therapies hearing; gene therapy; genetic therapies ... See more keywords

Genetic Therapies for Sickle Cell Disease.

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recommendations! Published in 2018 at "Pediatric clinics of North America"

DOI: 10.1016/j.pcl.2018.01.008

Abstract: Sickle cell disease is the most prevalent monogenic disorder worldwide and curative therapies are limited to hematopoietic stem cell transplant to the few with matched donors. Gene therapy has curative potential, whereby autologous hematopoietic stem… read more here.

Keywords: genetic therapies; cell disease; cell; therapies sickle ... See more keywords

Customizable mRNA Lipid Nanoparticles for Transfection of Primary Human T Cells.

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recommendations! Published in 2025 at "ACS nano"

DOI: 10.1021/acsnano.5c15903

Abstract: A central goal of genetic therapies, including RNA-based medicines, is to develop vectors that enable clinical translation for the treatment of specific cell types. T cells provide a unique opportunity for genetic medicines, particularly in… read more here.

Keywords: primary human; transfection primary; efficacy; human cells ... See more keywords

Duchenne muscular dystrophy: recent insights in brain related comorbidities

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recommendations! Published in 2025 at "Nature Communications"

DOI: 10.1038/s41467-025-56644-w

Abstract: Duchenne muscular dystrophy (DMD), the most common childhood muscular dystrophy, arises from DMD gene mutations, affecting the production of muscle dystrophin protein. Brain dystrophin-gene products are also transcribed via internal promoters. Their deficiency contributes to… read more here.

Keywords: duchenne muscular; brain; dystrophy recent; muscular dystrophy ... See more keywords

Genetic therapies for sickle cell disease.

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recommendations! Published in 2018 at "Seminars in hematology"

DOI: 10.1053/j.seminhematol.2018.04.014

Abstract: After decades with few novel therapeutic options for sickle cell disease (SCD), autologous hematopoietic stem cell (HSC) based genetic therapies including lentiviral gene therapy (GT), and genome editing (GE) now appear imminent. Lentiviral GT has… read more here.

Keywords: genetic therapies; cell disease; cell; therapies sickle ... See more keywords

Benefits and Shortcomings of Laboratory Model Systems in the Development of Genetic Therapies.

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recommendations! Published in 2022 at "Klinische Monatsblatter fur Augenheilkunde"

DOI: 10.1055/a-1757-9879

Abstract: Gene therapeutic approaches promise treatment or even a cure of diseases that were previously untreatable. Retinal gene therapies tested in clinical trials comprise a wide range of different strategies, including gene supplementation therapies, in vivo… read more here.

Keywords: genetic therapies; development genetic; benefits shortcomings; gene ... See more keywords

Advances in pharmacotherapy for sickle cell disease: what is the current state of play?

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recommendations! Published in 2024 at "Expert Opinion on Pharmacotherapy"

DOI: 10.1080/14656566.2024.2377711

Abstract: ABSTRACT Introduction Despite over 100 years of neglect and insufficient funding, sickle cell disease has risen to the top of the discussions due to the recent approval of two new genetic therapies. Prior to these approvals,… read more here.

Keywords: expert opinion; cell disease; cell; genetic therapies ... See more keywords

Biological and genetic therapies for the treatment of Duchenne muscular dystrophy

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recommendations! Published in 2022 at "Expert Opinion on Biological Therapy"

DOI: 10.1080/14712598.2022.2150543

Abstract: ABSTRACT Introduction Duchenne muscular dystrophy is a lethal genetic disease which currently has no cure, and poor standard treatment options largely focused on symptom relief. The development of multiple biological and genetic therapies is underway… read more here.

Keywords: duchenne muscular; therapy; biological genetic; muscular dystrophy ... See more keywords

Are Genetic Therapies for Epilepsy Ready for the Clinic?

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recommendations! Published in 2023 at "Epilepsy Currents"

DOI: 10.1177/15357597231176234

Abstract: In recent years, there has been a significant increase in preclinical studies to test genetic therapies for epilepsy. Some of these therapies have advanced to clinical trials and are being tested in patients with monogenetic… read more here.

Keywords: epilepsy ready; ready clinic; genetic therapies; therapies epilepsy ... See more keywords