Gene Therapy

The use of DNA as a technique for treating disease is a key feature of gene therapy. This type of treatment is being investigated to either replacement of a mutated gene with a healthy copy, silence a mutated gene that is dysfunctional or insert a novel gene to help fight disease. Discover the latest research on gene therapy here.

January 21, 2022

[Regulation effect of siRNA on β-globin in erythrocytes of hemoglobin H disease].

Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi
H L Cheng, R R Liu
January 21, 2022

Rapid characterization of adeno-associated virus (AAV) gene therapy vectors by mass photometry.

Gene Therapy
Di WuGrzegorz Piszczek
January 21, 2022
Comment / Editorial

Adeno-Associated Virus Delivery Limitations for Neurological Indications.

Human Gene Therapy
Hector Ribeiro Benatti, Heather L Gray-Edwards
January 21, 2022

Gene Therapy for Huntington's Disease: The Future Is in Gene Editing.

Human Gene Therapy
Ana Rita Batista
January 21, 2022

Astellas, Dyno to Develop Next-Generation Adeno-Associated Virus Vectors in Up-to-$1.6B Partnership.

Human Gene Therapy
Alex Philippidis
January 21, 2022

Acknowledgment of Reviewers 2021.

Human Gene Therapy
January 21, 2022
Review
Open Access

Gene Therapy Targeting PCSK9.

Metabolites
Julius L KatzmannUlrich Laufs
January 21, 2022

Design and characterization of a salicylic acid-inducible gene expression system for Jurkat cells.

Journal of Biotechnology
Aarti DoshiPatrick C Cirino
January 21, 2022

Genome DNA Leakage of Adeno-Associated Virus Under Freeze-Thaw Stress.

International Journal of Pharmaceutics
Alex Yuechuan XuDingjiang Liu
January 20, 2022

Nuclear and mitochondrial DNA editing in human cells with zinc finger deaminases.

Nature Communications
Kayeong LimJin-Soo Kim
January 20, 2022

Risk factors and abnormal cerebrospinal fluid associate with cognitive symptoms after mild COVID-19.

Annals of Clinical and Translational Neurology
Alexandra C AppleJoanna Hellmuth
January 20, 2022

Loss-of-function mutations in the co-chaperone protein BAG5 cause dilated cardiomyopathy requiring heart transplantation.

Science Translational Medicine
Hideyuki HakuiYoshihiro Asano
January 20, 2022

The neuronal retromer can regulate both neuronal and microglial phenotypes of Alzheimer's disease.

Cell Reports
Yasir H QureshiScott A Small
January 19, 2022

Decoding the Kinetic Pathways toward a Lipid/DNA Complex of Alkyl Alcohol Cationic Lipids Formed in a Microfluidic Channel.

The Journal of Physical Chemistry. B
Dipanjan MukherjeeSamir Kumar Pal
January 19, 2022

Targeting PTPN22 does not enhance the efficacy of CAR T cells in solid tumours.

Molecular and Cellular Biology
Xin DuTony Tiganis
January 19, 2022
Open Access

Therapeutic potential of highly functional codon-optimized microutrophin for muscle-specific expression.

Scientific Reports
Anna V StarikovaMaryana V Bardina
January 18, 2022

Preparation and characterization of polyamidoamine dendrimers conjugated with cholesteryl-dipeptide as gene carriers in HeLa cells.

Journal of Biomaterials Science. Polymer Edition
Le Thi ThuyJoon Sig Choi

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