Volume 30 Issue 1-2, February 2023

Cover credit: Purkinje cells transduced by AAV9 following intracisternal delivery in rats. The image shows a rat Purkinje cell transduced by a single-stranded AAV9-EGFP delivered to the cisterna magna next to the untransduced granule cell layer stained by Neu N.

Review Article

Progress on siRNA-based gene therapy targeting secondary injury after intracerebral hemorrhage

Daniyah A. Almarghalani
Zahoor A. Shah
Review Article 10 Nov 2021
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Curing SMA: Are we there yet?

Aoife Reilly
Lucia Chehade
Rashmi Kothary
Review Article 26 May 2022

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Article

Intracerebral lentiviral ABCD1 gene therapy in an early disease onset ALD mouse model

Jie Gong
Yunyun Liu
Lung-Ji Chang

Collection:

Editor's Choice
Article 06 Jul 2022
Effect of nanoparticle-mediated delivery of SFRP4 siRNA for treating Dupuytren disease

Rujue Jin
Weigang Zhu
Aidong Deng
Article 28 Mar 2022
Adjuvant properties of IFN-γ and GM-CSF in the scFv6.C4 DNA vaccine against CEA-expressing tumors

Bianca Ferrarini Zanetti
Camila Pontes Ferreira
Sang Won Han
Article 09 Jun 2021
E3 ubiquitin ligase SMURF2 prevents colorectal cancer by reducing the stability of the YY1 protein and inhibiting the SENP1/c-myc axis

Qianfu Gao
Shanchao Wang
Zeyan Zhang
Article 21 Sept 2021
Oncolytic Newcastle disease virus expressing the co-stimulator OX40L as immunopotentiator for colorectal cancer therapy

Limin Tian
Tianyan Liu
Deshan Li
Article 04 Oct 2021
Overexpressed ski efficiently promotes neurorestoration, increases neuronal regeneration, and reduces astrogliosis after traumatic brain injury

Yu Zhai
Shi-Yang Ye
Yuan-Guo Zhou
Article 08 Feb 2022
Exosomal PD-L1 confers chemoresistance and promotes tumorigenic properties in esophageal cancer cells via upregulating STAT3/miR-21

Haojie Wang
Yijun Qi
Gaofeng Liang
Article 19 Apr 2022
Adeno-associated virus serotype 9 antibody titers in patients with SMA pre-screened for treatment with onasemnogene abeparvovec –routine care evidence

Sharon Aharoni
Jacob Bistritzer
Yoram Nevo
Article 27 Apr 2022
Efficient CRISPR/Cas9 nickase-mediated genome editing in an in vitro model of mucopolysaccharidosis IVA

Andrés Felipe Leal
Carlos Javier Alméciga-Díaz
Article 18 May 2022
Effects of intracardiac delivery of aldehyde dehydrogenase 2 gene in myocardial salvage

Guodong Pan
Bipradas Roy
Suresh Selvaraj Palaniyandi
Article 24 May 2022
Enzymatically amplified linear dbDNA^TM as a rapid and scalable solution to industrial lentiviral vector manufacturing

Maria Barreira
Claire Kerridge
Sarah Smith-Moore
Article Open Access 24 May 2022
Assessment of AAV9 distribution and transduction in rats after administration through Intrastriatal, Intracisterna magna and Lumbar Intrathecal routes

Jayanth Chandran
Ekram Ahmed Chowdhury
Guy M. L. Meno-Tetang
Article 30 May 2022
Forced activation of dystrophin transcription by CRISPR/dCas9 reduced arrhythmia susceptibility via restoring membrane Nav1.5 distribution

Ruixin Zhang
Junwu Liu
Zhenwei Pan
Article 30 May 2022
Clinical enrollment assay to detect preexisting neutralizing antibodies to AAV6 with demonstrated transgene expression in gene therapy trials

Liching Cao
Annemarie Ledeboer
Kathleen Meyer
Article Open Access 01 Jul 2022
Identification of RPGR ORF15 mutation for X-linked retinitis pigmentosa in a large Chinese family and in vitro correction with prime editor

Xiujuan Lv
Zheng Zheng
Feng Gu
Article 06 Jul 2022

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Brief Communication

Easy and robust electrotransfection protocol for efficient ectopic gene expression and genome editing in human B cells

Reynand Jay Canoy
Franck André
Diego Germini

Collection:

Gene Editing
Brief Communication 30 Sept 2020
Host antibacterial defense of 6–10 Gy γ-irradiated mice subjected to lentiviral vector-based Gas5 gene therapy

Ichiaki Ito
Bradford D. Loucas
Fujio Suzuki
Brief Communication 01 Dec 2020

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Perspective

Equitable access to cell and gene therapies in South Africa: opportunities and hurdles

Candice Laverne Hendricks
Marco Alessandrini
Michael Sean Pepper
Perspective 08 Jan 2022

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Correction

Correction: Optimisation of a TALE nuclease targeting the HIV co-receptor CCR5 for clinical application

Lea Isabell Schwarze
Dawid Głów
Boris Fehse
Correction Open Access 22 Nov 2022
Correction: Systematic review and meta-analysis determining the benefits of in vivo genetic therapy in spinal muscular atrophy rodent models

Ellie M. Chilcott
Evalyne W. Muiruri
Rafael J. Yáñez-Muñoz
Correction Open Access 28 Nov 2022

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Volume 30 Issue 1-2, February 2023

Review Article

Progress on siRNA-based gene therapy targeting secondary injury after intracerebral hemorrhage

Curing SMA: Are we there yet?

Article

Intracerebral lentiviral ABCD1 gene therapy in an early disease onset ALD mouse model

Effect of nanoparticle-mediated delivery of SFRP4 siRNA for treating Dupuytren disease

Adjuvant properties of IFN-γ and GM-CSF in the scFv6.C4 DNA vaccine against CEA-expressing tumors

E3 ubiquitin ligase SMURF2 prevents colorectal cancer by reducing the stability of the YY1 protein and inhibiting the SENP1/c-myc axis

Oncolytic Newcastle disease virus expressing the co-stimulator OX40L as immunopotentiator for colorectal cancer therapy

Overexpressed ski efficiently promotes neurorestoration, increases neuronal regeneration, and reduces astrogliosis after traumatic brain injury

Exosomal PD-L1 confers chemoresistance and promotes tumorigenic properties in esophageal cancer cells via upregulating STAT3/miR-21

Adeno-associated virus serotype 9 antibody titers in patients with SMA pre-screened for treatment with onasemnogene abeparvovec –routine care evidence

Efficient CRISPR/Cas9 nickase-mediated genome editing in an in vitro model of mucopolysaccharidosis IVA

Effects of intracardiac delivery of aldehyde dehydrogenase 2 gene in myocardial salvage

Enzymatically amplified linear dbDNA^TM as a rapid and scalable solution to industrial lentiviral vector manufacturing

Assessment of AAV9 distribution and transduction in rats after administration through Intrastriatal, Intracisterna magna and Lumbar Intrathecal routes

Forced activation of dystrophin transcription by CRISPR/dCas9 reduced arrhythmia susceptibility via restoring membrane Nav1.5 distribution

Clinical enrollment assay to detect preexisting neutralizing antibodies to AAV6 with demonstrated transgene expression in gene therapy trials

Identification of RPGR ORF15 mutation for X-linked retinitis pigmentosa in a large Chinese family and in vitro correction with prime editor

Brief Communication

Easy and robust electrotransfection protocol for efficient ectopic gene expression and genome editing in human B cells

Host antibacterial defense of 6–10 Gy γ-irradiated mice subjected to lentiviral vector-based Gas5 gene therapy

Perspective

Equitable access to cell and gene therapies in South Africa: opportunities and hurdles

Correction

Correction: Optimisation of a TALE nuclease targeting the HIV co-receptor CCR5 for clinical application

Correction: Systematic review and meta-analysis determining the benefits of in vivo genetic therapy in spinal muscular atrophy rodent models

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