Research Progress of nucleic acid delivery vectors for gene therapy

Jiao, Yang; Xia, Zhang Li; Ze, Li Jiang; Jing, Hui; Bai, Xin; Sun, Fan

doi:10.1007/s10544-020-0469-7

Cited by 36 publications

(23 citation statements)

References 42 publications

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“…This study confirmed that knockdown of AP-2α restricted the expression of TGF-β1 and Smad3 to promote proliferation and reduce the senescence and apoptosis of NP cells in rats with IVDD [59]. AAVs could be effective tools for gene therapy [60,61].…”

Section: Adeno-associated Virus (Aav)supporting

confidence: 71%

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Genetic Therapy for Intervertebral Disc Degeneration

Roh

Darai

Kyung

et al. 2021

IJMS

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Intervertebral disc (IVD) degeneration can cause chronic lower back pain (LBP), leading to disability. Despite significant advances in the treatment of discogenic LBP, the limitations of current treatments have sparked interest in biological approaches, including growth factor and stem cell injection, as new treatment options for patients with chronic LBP due to IVD degeneration (IVDD). Gene therapy represents exciting new possibilities for IVDD treatment, but treatment is still in its infancy. Literature searches were conducted using PubMed and Google Scholar to provide an overview of the principles and current state of gene therapy for IVDD. Gene transfer to degenerated disc cells in vitro and in animal models is reviewed. In addition, this review describes the use of gene silencing by RNA interference (RNAi) and gene editing by the clustered regularly interspaced short palindromic repeats (CRISPR) system, as well as the mammalian target of rapamycin (mTOR) signaling in vitro and in animal models. Significant technological advances in recent years have opened the door to a new generation of intradiscal gene therapy for the treatment of chronic discogenic LBP.

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Section: Adeno-associated Virus (Aav)supporting

confidence: 71%

“…In 2020, Bi et al [64] attenuated H reduce the senescence and apoptosis of NP cells in rats with IVDD [59]. AAVs could be effective tools for gene therapy [60,61].…”

Section: Non-virus Vector-mediated Gene Transfer To Disc Cells Rna Interference (Rnai)mentioning

confidence: 99%

Genetic Therapy for Intervertebral Disc Degeneration

Roh

Darai

Kyung

et al. 2021

IJMS

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“…206 However, there are also some disadvantages to use viral vectors, including immunogenicity and potential risk of inducing mutations, making viral vectors potential safety hazards. 207 Adeno-associated virus-mediated delivery of shRNAs in normal rats resulted in a 35% knockdown of α-synuclein without affecting motor function or causing degeneration of dopaminergic neurons. 208 The α-synuclein knockdown by shRNAs in a PD rat model was observed to be neuroprotective, decreasing the degeneration of dopaminergic neurons and attenuating the progression of motor deficits.…”

Section: Short Hairpin Rnasmentioning

confidence: 99%

“…Since host cells can continuously synthesize shRNAs, they have several advantages over siRNA including longer‐lasting effects, lower dose requirement, and less specific and nonspecific off‐target effects 206 . However, there are also some disadvantages to use viral vectors, including immunogenicity and potential risk of inducing mutations, making viral vectors potential safety hazards 207 . Adeno‐associated virus‐mediated delivery of shRNAs in normal rats resulted in a 35% knockdown of α‐synuclein without affecting motor function or causing degeneration of dopaminergic neurons 208 .…”

Section: Nucleic Acid Therapeutics and Its Progress In Pd Researchmentioning

confidence: 99%

Progress in the molecular pathogenesis and nucleic acid therapeutics for Parkinson's disease in the precision medicine era

Mastaglia

Fletcher

et al. 2020

Medicinal Research Reviews

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Parkinson's disease (PD) is one of the most common neurodegenerative disorders that manifest various motor and nonmotor symptoms. Although currently available therapies can alleviate some of the symptoms, the disease continues to progress, leading eventually to severe motor and cognitive decline and reduced life expectancy. The past two decades have witnessed rapid progress in our understanding of the molecular and genetic pathogenesis of the disease, paving the way for the development of new therapeutic approaches to arrest or delay the neurodegenerative process. As a result of these advances, biomarker-driven subtyping is making it possible to stratify PD patients into more homogeneous subgroups that may better respond to potential genetic-molecular pathway targeted disease-modifying therapies. Therapeutic nucleic acid oligomers can bind to target gene sequences with very high specificity in a base-pairing manner and precisely modulate downstream molecular events. Recently, nucleic acid therapeutics have proven effective in the treatment of a number of severe neurological and neuromuscular disorders, drawing increasing attention to the possibility of developing novel molecular therapies for PD. In this

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“…All these properties are especially relevant when carrying out in vivo magnetofection. There are a number of reviews in the literature that are dedicated to various aspects of the nonviral delivery of genetic information, such as the properties and characteristics of vectors for in vitro magnetofection [ 9 , 47 , 52 , 53 , 54 , 55 , 56 , 57 ], the mechanism and distinctive features of transfection (magnetofection) [ 9 , 26 , 47 , 49 , 51 , 52 , 56 , 57 , 58 , 59 , 60 , 61 , 62 , 63 ], in vivo [ 8 , 26 , 51 , 58 , 59 , 61 , 64 , 65 , 66 , 67 , 68 , 69 , 70 , 71 , 72 ] and in vitro [ 8 , 26 , 51 , 61 , 64 , 65 , 67 , 69 , 71 , 72 , 73 ,…”

Section: Introductionmentioning

confidence: 99%

Nonviral Locally Injected Magnetic Vectors for In Vivo Gene Delivery: A Review of Studies on Magnetofection

et al. 2021

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Magnetic nanoparticles have been widely used in nanobiomedicine for diagnostics and the treatment of diseases, and as carriers for various drugs. The unique magnetic properties of “magnetic” drugs allow their delivery in a targeted tumor or tissue upon application of a magnetic field. The approach of combining magnetic drug targeting and gene delivery is called magnetofection, and it is very promising. This method is simple and efficient for the delivery of genetic material to cells using magnetic nanoparticles controlled by an external magnetic field. However, magnetofection in vivo has been studied insufficiently both for local and systemic routes of magnetic vector injection, and the relevant data available in the literature are often merely descriptive and contradictory. In this review, we collected and systematized the data on the efficiency of the local injections of magnetic nanoparticles that carry genetic information upon application of external magnetic fields. We also investigated the efficiency of magnetofection in vivo, depending on the structure and coverage of magnetic vectors. The perspectives of the development of the method were also considered.

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Research Progress of nucleic acid delivery vectors for gene therapy

Cited by 36 publications

References 42 publications

Genetic Therapy for Intervertebral Disc Degeneration

Genetic Therapy for Intervertebral Disc Degeneration

Progress in the molecular pathogenesis and nucleic acid therapeutics for Parkinson's disease in the precision medicine era

Nonviral Locally Injected Magnetic Vectors for In Vivo Gene Delivery: A Review of Studies on Magnetofection

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