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oa

Serpin B9-Insensitive Granzyme B Mutant Delivered by Engineered Capsid AAV Vectors Demonstrates Selective Killing of EGFR-Positive Cancer Cells
Dennis Makafui Dogbey, Krupa Naran, Stefan Barth,

First published: 13 June 2025
Research Article
Open Access

oa

Long-Term Correction of Murine Glycogen Storage Disease Type III by AAV-Mediated Gene Therapy Using an Immunotolerizing Dual Promoter to Express Bacterial Pullulanase
Kuo-An Liao, Jeong-A Lim, Su Jin Choi, Haiqing Yi, Baodong Sun,

First published: 17 April 2025
Research Article
Open Access

oa

The Impact of Niacin Administration on Plasma Lipids and Gene Expression in the Vervet Monkey Model (Chlorocebus aethiops)
Thobile Ngqaneka, Zandisiwe Emilia Magwebu, Kenechukwu Obikeze, Chesa Gift Chauke,

First published: 05 February 2025
Research Article
Open Access

oa

Silencing of B4Galnt1 Gene Prevents GM2 Accumulation in Tay-Sachs Cells
Nurselin Ateş, Orhan Kerim İnci, Seçil Akyıldız Demir, Volkan Seyrantepe,

First published: 26 March 2024
Research Article
Open Access

oa

Induction of Exocytosis Rescues Lysosomal GM2 Accumulation in Tay-Sachs Disease
Nurselin Ateş, Secil Akyildiz Demir, Volkan Seyrantepe,

First published: 19 March 2024
Research Article
Open Access

oa

Comprehensive Comparison of AAV Purification Methods: Iodixanol Gradient Centrifugation vs. Immuno-Affinity Chromatography
Anh K. Lam, Patrick L. Mulcrone, Dylan Frabutt, Junping Zhang, Matthew Chrzanowski, Sreevani Arisa, Maite Munoz, Xin Li, Moanaro Biswas, David Markusic, Roland W. Herzog, Weidong Xiao,

First published: 11 December 2023

Collections: Viral Vectors and their Application in Treating Inherited Diseases
Research Article
Open Access

oa

Therapeutic Effect of Hydrodynamics-Based Delivery of Matrix Metalloproteinase-13 Gene on Thioacetamide-Induced Liver Fibrosis in Rats
Takeshi Yokoo, Kenya Kamimura, Ryosuke Nozawa, Moeno Sugita, Osamu Shibata, Yuji Kobayashi, Hiroyuki Abe, Hiromi Miura, Masato Ohtsuka, Shuji Terai,

First published: 11 October 2023

Collections: Development of Novel Nonviral Gene Therapy Technologies
Research Article
Open Access

oa

Intra-Articular AAV9 α-l-Iduronidase Gene Replacement in the Canine Model of Mucopolysaccharidosis Type I
Raymond Yu-Jeang Wang, Shih-Hsin Kan, Haoyue Zhang, Jodi D. Smith, Afshin Aminian, Elizabeth Snella, Jackie K. Jens, Sarah P. Young, Patricia I. Dickson, N. Matthew Ellinwood,

First published: 14 September 2023

Collections: Viral Vectors and their Application in Treating Inherited Diseases
Review Article
Open Access

oa

Diagnostic and Therapeutic Application of Proteomics in Infectious Disease
Fanuel Bizuayehu Yihunie, Mequanint Addisu Belete, Gizachew Fentahun, Solomon Getachew, Teshager Dubie,

First published: 24 August 2023
Research Article
Open Access

oa

The Association between Genetic Variants and Gene Expression in RAAS Genes Using Captive-Bred Vervet Monkeys (Chlorocebus aethiops)
Sanele Khoza, Meenu Ghai, Chesa G. Chauke, Zandisiwe E. Magwebu,

First published: 17 February 2023
Review Article
Open Access

oa

Vaccine Boosting CAR-T Cell Therapy: Current and Future Strategies
Emmanuel Owusu Ansah, Andy Baah, Emmanuel Boateng Agyenim,

First published: 31 January 2023
Research Article
Open Access

oa

Intravitreally Administered Soluble VEGF Receptor-1 Variant Tested as a Potential Gene Therapeutic for Diabetic Retinopathy
Steven Hyun Seung Lee, Joo Yong Lee, Im Kyeung Kang, Jun-Sub Choi, Hee Jong Kim, Jin Kim, Seho Cha, Kyoung Jin Lee, Ha-Na Woo, Keerang Park, Heuiran Lee,

First published: 13 October 2022
Review Article
Open Access

oa

Ethical Challenges and Controversies in the Practice and Advancement of Gene Therapy
Emmanuel Owusu Ansah,

First published: 24 August 2022
Research Article
Open Access

oa

Inadvertent Transfer of Murine VL30 Retrotransposons to CAR-T Cells
Sung Hyun Lee, Yajing Hao, Tong Gui, Gianpietro Dotti, Barbara Savoldo, Fei Zou, Tal Kafri,

First published: 31 May 2022
Research Article
Open Access

oa

Genomic Designs of rAAVs Contribute to Pathological Changes in the Livers and Spleens of Mice
Patrick L. Mulcrone, Junping Zhang, P. Melanie Pride, Anh K. Lam, Dylan A. Frabutt, Susan M. Ball-Kell, Weidong Xiao,

First published: 20 March 2022
ISSUE INFORMATION
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free

Issue Information
First published: 20 October 2021
- Abstract
- PDF
ORIGINAL ARTICLE
Full Access

full

Identification of homologous human miRNAs as antivirals towards COVID-19 genome
Jitender Singh, Ashvinder Raina, Namrata Sangwan, Arushi Chauhan, Krishan L. Khanduja, Pramod K. Avti,

First published: 13 September 2021

Graphical Abstract
ISSUE INFORMATION
Free Access

free

Issue Information
First published: 26 July 2021
- First Page
- PDF
INVITED REVIEW
Open Access

oa

Immunological tumor heterogeneity and diagnostic profiling for advanced and immune therapies
Ralf Huss, Christoph Schmid, Mael Manesse, Jeppe Thagaard, Bruno Maerkl,

First published: 25 June 2021
LETTER TO THE EDITOR
Full Access

full

Inhospital morbidity and mortality among acute myeloid leukemia patients admitted for hematopoietic stem cell transplantation between 2002-2017
Saqib Abbasi, Joseph Bennett, Osama Diab, Ghulam Rehman Mohyuddin, Anurag Singh,

First published: 15 May 2021

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Advances in Cell and Gene Therapy

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All Advances in Cell and Gene Therapy Articles

oa

Serpin B9-Insensitive Granzyme B Mutant Delivered by Engineered Capsid AAV Vectors Demonstrates Selective Killing of EGFR-Positive Cancer Cells

oa

Long-Term Correction of Murine Glycogen Storage Disease Type III by AAV-Mediated Gene Therapy Using an Immunotolerizing Dual Promoter to Express Bacterial Pullulanase

oa

The Impact of Niacin Administration on Plasma Lipids and Gene Expression in the Vervet Monkey Model (Chlorocebus aethiops)

oa

Silencing of B4Galnt1 Gene Prevents GM2 Accumulation in Tay-Sachs Cells

oa

Induction of Exocytosis Rescues Lysosomal GM2 Accumulation in Tay-Sachs Disease

oa

Comprehensive Comparison of AAV Purification Methods: Iodixanol Gradient Centrifugation vs. Immuno-Affinity Chromatography

oa

Therapeutic Effect of Hydrodynamics-Based Delivery of Matrix Metalloproteinase-13 Gene on Thioacetamide-Induced Liver Fibrosis in Rats

oa

Intra-Articular AAV9 α-l-Iduronidase Gene Replacement in the Canine Model of Mucopolysaccharidosis Type I

oa

Diagnostic and Therapeutic Application of Proteomics in Infectious Disease

oa

The Association between Genetic Variants and Gene Expression in RAAS Genes Using Captive-Bred Vervet Monkeys (Chlorocebus aethiops)

oa

Vaccine Boosting CAR-T Cell Therapy: Current and Future Strategies

oa

Intravitreally Administered Soluble VEGF Receptor-1 Variant Tested as a Potential Gene Therapeutic for Diabetic Retinopathy

oa

Ethical Challenges and Controversies in the Practice and Advancement of Gene Therapy

oa

Inadvertent Transfer of Murine VL30 Retrotransposons to CAR-T Cells

oa

Genomic Designs of rAAVs Contribute to Pathological Changes in the Livers and Spleens of Mice

free

Issue Information

full

Identification of homologous human miRNAs as antivirals towards COVID-19 genome

Graphical Abstract

free

Issue Information

oa

Immunological tumor heterogeneity and diagnostic profiling for advanced and immune therapies

full

Inhospital morbidity and mortality among acute myeloid leukemia patients admitted for hematopoietic stem cell transplantation between 2002-2017

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oa Serpin B9-Insensitive Granzyme B Mutant Delivered by Engineered Capsid AAV Vectors Demonstrates Selective Killing of EGFR-Positive Cancer Cells

oa Long-Term Correction of Murine Glycogen Storage Disease Type III by AAV-Mediated Gene Therapy Using an Immunotolerizing Dual Promoter to Express Bacterial Pullulanase

oa The Impact of Niacin Administration on Plasma Lipids and Gene Expression in the Vervet Monkey Model (Chlorocebus aethiops)

oa Silencing of B4Galnt1 Gene Prevents GM2 Accumulation in Tay-Sachs Cells

oa Induction of Exocytosis Rescues Lysosomal GM2 Accumulation in Tay-Sachs Disease

oa Comprehensive Comparison of AAV Purification Methods: Iodixanol Gradient Centrifugation vs. Immuno-Affinity Chromatography

oa Therapeutic Effect of Hydrodynamics-Based Delivery of Matrix Metalloproteinase-13 Gene on Thioacetamide-Induced Liver Fibrosis in Rats

oa Intra-Articular AAV9 α-l-Iduronidase Gene Replacement in the Canine Model of Mucopolysaccharidosis Type I

oa Diagnostic and Therapeutic Application of Proteomics in Infectious Disease

oa The Association between Genetic Variants and Gene Expression in RAAS Genes Using Captive-Bred Vervet Monkeys (Chlorocebus aethiops)

oa Vaccine Boosting CAR-T Cell Therapy: Current and Future Strategies

oa Intravitreally Administered Soluble VEGF Receptor-1 Variant Tested as a Potential Gene Therapeutic for Diabetic Retinopathy

oa Ethical Challenges and Controversies in the Practice and Advancement of Gene Therapy

oa Inadvertent Transfer of Murine VL30 Retrotransposons to CAR-T Cells

oa Genomic Designs of rAAVs Contribute to Pathological Changes in the Livers and Spleens of Mice

free Issue Information

full Identification of homologous human miRNAs as antivirals towards COVID-19 genome

Graphical Abstract

free Issue Information

oa Immunological tumor heterogeneity and diagnostic profiling for advanced and immune therapies

full Inhospital morbidity and mortality among acute myeloid leukemia patients admitted for hematopoietic stem cell transplantation between 2002-2017

oa

Serpin B9-Insensitive Granzyme B Mutant Delivered by Engineered Capsid AAV Vectors Demonstrates Selective Killing of EGFR-Positive Cancer Cells

oa

Long-Term Correction of Murine Glycogen Storage Disease Type III by AAV-Mediated Gene Therapy Using an Immunotolerizing Dual Promoter to Express Bacterial Pullulanase

oa

The Impact of Niacin Administration on Plasma Lipids and Gene Expression in the Vervet Monkey Model (Chlorocebus aethiops)

oa

Silencing of B4Galnt1 Gene Prevents GM2 Accumulation in Tay-Sachs Cells

oa

Induction of Exocytosis Rescues Lysosomal GM2 Accumulation in Tay-Sachs Disease

oa

Comprehensive Comparison of AAV Purification Methods: Iodixanol Gradient Centrifugation vs. Immuno-Affinity Chromatography

oa

Therapeutic Effect of Hydrodynamics-Based Delivery of Matrix Metalloproteinase-13 Gene on Thioacetamide-Induced Liver Fibrosis in Rats

oa

Intra-Articular AAV9 α-l-Iduronidase Gene Replacement in the Canine Model of Mucopolysaccharidosis Type I

oa

Diagnostic and Therapeutic Application of Proteomics in Infectious Disease

oa

The Association between Genetic Variants and Gene Expression in RAAS Genes Using Captive-Bred Vervet Monkeys (Chlorocebus aethiops)

oa

Vaccine Boosting CAR-T Cell Therapy: Current and Future Strategies

oa

Intravitreally Administered Soluble VEGF Receptor-1 Variant Tested as a Potential Gene Therapeutic for Diabetic Retinopathy

oa

Ethical Challenges and Controversies in the Practice and Advancement of Gene Therapy

oa

Inadvertent Transfer of Murine VL30 Retrotransposons to CAR-T Cells

oa

Genomic Designs of rAAVs Contribute to Pathological Changes in the Livers and Spleens of Mice

free

Issue Information

full

Identification of homologous human miRNAs as antivirals towards COVID-19 genome

free

Issue Information

oa

Immunological tumor heterogeneity and diagnostic profiling for advanced and immune therapies

full

Inhospital morbidity and mortality among acute myeloid leukemia patients admitted for hematopoietic stem cell transplantation between 2002-2017