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Article ; Online: Recent advances in hematopoietic gene therapy for genetic disorders.

Galy, Anne / Dewannieux, Marie

Archives de pediatrie : organe officiel de la Societe francaise de pediatrie

2023 Volume 30, Issue 8S1, Page(s) 8S24–8S31

Abstract: Hematopoietic gene therapy is based on the transplantation of gene-modified autologous hematopoietic stem cells and since the inception of this approach, many technological and medical improvements have been achieved. This review focuses on the clinical ... ...

Abstract	Hematopoietic gene therapy is based on the transplantation of gene-modified autologous hematopoietic stem cells and since the inception of this approach, many technological and medical improvements have been achieved. This review focuses on the clinical studies that have used hematopoietic gene therapy to successfully treat several rare and severe genetic disorders of the blood or immune system as well as some non-hematological diseases. Today, in some cases hematopoietic gene therapy has progressed to the point of being equal to, or better than, allogeneic bone marrow transplant. In others, further improvements are needed to obtain more consistent efficacy or to reduce the risks posed by vectors or protocols. Several hematopoietic gene therapy products showing both long-term efficacy and safety have reached the market, but economic considerations challenge the possibility of patient access to novel disease-modifying therapies. © 2023 Published by Elsevier Masson SAS on behalf of French Society of Pediatrics.
MeSH term(s)	Child ; Humans ; Hematopoietic Stem Cell Transplantation ; Hematopoietic Stem Cells ; Genetic Therapy/methods
Language	English
Publishing date	2023-10-17
Publishing country	France
Document type	Review ; Journal Article
ZDB-ID	1181947-9
ISSN	1769-664X ; 0929-693X
ISSN (online)	1769-664X
ISSN	0929-693X
DOI	10.1016/S0929-693X(23)00224-5
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Zs.A 3942: Show issues

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Article: Evaluation of diversity indices to estimate clonal dominance in gene therapy studies.

Corre, Guillaume / Galy, Anne

Molecular therapy. Methods & clinical development

2023 Volume 29, Page(s) 418–425

Abstract: In cell and gene therapy, achieving the stable engraftment of an abundant and highly polyclonal population of gene-corrected cells is one of the key factors to ensure the successful and safe treatment of patients. Because integrative vectors have been ... ...

Abstract	In cell and gene therapy, achieving the stable engraftment of an abundant and highly polyclonal population of gene-corrected cells is one of the key factors to ensure the successful and safe treatment of patients. Because integrative vectors have been associated with possible risks of insertional mutagenesis leading to clonal dominance, monitoring the relative abundance of individual vector insertion sites in patients' blood cells has become an important safety assessment, particularly in hematopoietic stem cell-based therapies. Clinical studies often express clonal diversity using various metrics. One of the most commonly used is the Shannon index of entropy. However, this index aggregates two distinct aspects of diversity, the number of unique species and their relative abundance. This property hampers the comparison of samples with different richness. This prompted us to reanalyze published datasets and to model the properties of various indices as applied to the evaluation of clonal diversity in gene therapy. A normalized version of the Shannon index, such as Pielou's index, or Simpson's probability index is robust and useful to compare sample evenness between patients and trials. Clinically meaningful standard values for clonal diversity are herein proposed to facilitate the use of vector insertion site analyses in genomic medicine practice.
Language	English
Publishing date	2023-05-09
Publishing country	United States
Document type	Journal Article
ZDB-ID	2872938-9
ISSN	2329-0501 ; 2329-0501
ISSN (online)	2329-0501
ISSN	2329-0501
DOI	10.1016/j.omtm.2023.05.003
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Zs.A 7107: Show issues

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Article ; Online: Major Advances in the Development of Vectors for Clinical Gene Therapy of Hematopoietic Stem Cells from European Groups over the Last 25 Years.

Galy, Anne

Human gene therapy

2017 Volume 28, Issue 11, Page(s) 964–971

Abstract: The first attempts at hematopoietic stem cell-based gene therapy (HSC-GT) were reported >25 years ago for primary immune deficiencies, marking the beginning of a vibrant field of translational and therapeutic research. Since then, many HSC-GT studies ... ...

Abstract	The first attempts at hematopoietic stem cell-based gene therapy (HSC-GT) were reported >25 years ago for primary immune deficiencies, marking the beginning of a vibrant field of translational and therapeutic research. Since then, many HSC-GT studies have been conducted in diverse genetic diseases. The approach has been improved over time, showing biological and therapeutic efficacy with an overall excellent safety record. Within a defined regulatory and ethical landscape, the field of HSC-GT has reached industrialization and commercialization stages, with a landmark recent approval by the European Medicines Agency of the first HSC-GT medicine for human use. At such a pivotal stage, it is important to look back at 25 years of European applied research in this field. This review highlights some of the key contributions of European teams to the field of HSC-GT, focusing in particular on the development of safer gene transfer vectors and international cooperation.
MeSH term(s)	Genetic Therapy/trends ; Genetic Vectors/therapeutic use ; Hematopoietic Stem Cell Transplantation ; Hematopoietic Stem Cells/cytology ; Humans ; Retroviridae/genetics
Language	English
Publishing date	2017-08-12
Publishing country	United States
Document type	Journal Article ; Review
ZDB-ID	1028152-6
ISSN	1557-7422 ; 1043-0342
ISSN (online)	1557-7422
ISSN	1043-0342
DOI	10.1089/hum.2017.152
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Zs.A 2988: Show issues

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Article ; Online: Like Angler Fish, CAARs Lure Their Prey.

Galy, Anne

Molecular therapy : the journal of the American Society of Gene Therapy

2016 Volume 24, Issue 8, Page(s) 1339–1341

Language	English
Publishing date	2016-08
Publishing country	United States
Document type	Journal Article
ZDB-ID	2010592-7
ISSN	1525-0024 ; 1525-0016
ISSN (online)	1525-0024
ISSN	1525-0016
DOI	10.1038/mt.2016.165
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Zs.A 5640: Show issues

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Book ; Online: Immune responses to AAV vectors, from bench to bedside

Tschakarjan, Etiena Basner / Buning, Hildegard / Mingozzi, Federico / Galy, Anne

2015

Abstract: The recent wave of clinical studies demonstrating long-term therapeutic efficacy highlights the enormous potential of gene therapy as an approach to the treatment of inherited disorders and cancer. While in recent years lentiviral vectors have dominated ... ...

Abstract	The recent wave of clinical studies demonstrating long-term therapeutic efficacy highlights the enormous potential of gene therapy as an approach to the treatment of inherited disorders and cancer. While in recent years lentiviral vectors have dominated the field of ex vivo gene therapy in man, adeno-associated virus (AAV) vectors have become the platform of choice for the in vivo gene delivery, both local and systemic.Despite the achievements in the clinic however, a number of hurdles remain to be overcome in gene therapy, these include availability of scalable vector production systems, potential issues associated with insertional mutagenesis, and concerns related to immunogenicity of gene therapeutics. For AAV vectors, clinical trials showed that immunity directed against the vector could either prevent transduction of a target tissue or limit the duration of therapeutic efficacy.- Initial observations in the context of a gene therapy trial for hemophilia spurred over a decade efforts by gene therapists and immunologists to understand the mechanism and identify factors that contribute to AAV's immunogenicity, including the prevalence of B cell and T cell immunity to wild type AAV in humans and the interaction of AAV vectors with the innate and adaptive immune system. Despite a number of important contributions in particular in the more recent past, our knowledge on the immunology of gene transfer is still rudimental; this is partly due to the fact that the basic understanding of the complex balance between tolerance and immunity to an antigen, key aspect of gene transfer with AAV, keeps evolving rapidly.- However, continuing work towards a better definition of the interaction of viral vectors with the immune system has led to significant advances in the knowledge of the factors influencing the outcome of gene transfer, such as the vector dose, the immune privilege of certain tissues, and the induction of tolerance to an antigen. A better understanding of the structure-function relationship of the viral capsid has boosted the development of novel immune-escape vector variants. In addition, novel immunomodulatory strategies were established to prevent or reduce anti-capsid immunity have been developed and are being tested in preclinical models and in clinical trials. Together, these advances are bringing us closer to the goal of achieving safe and sustained therapeutic gene transfer in humans.-
Keywords	Immunologic diseases. Allergy ; Medicine (General)
Size	1 electronic resource (95 p.)
Publisher	Frontiers Media SA
Document type	Book ; Online
Note	English ; Open Access
HBZ-ID	HT020090107
ISBN	9782889195008 ; 2889195007
Database	ZB MED Catalogue: Medicine, Health, Nutrition, Environment, Agriculture

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Article ; Online: Hematopoietic stem and progenitors cells gene editing: Beyond blood disorders.

Buffa, Valentina / Alvarez Vargas, José Roberto / Galy, Anne / Spinozzi, Simone / Rocca, Céline J

Frontiers in genome editing

2023 Volume 4, Page(s) 997142

Abstract: Lessons learned from decades-long practice in the transplantation of hematopoietic stem and progenitor cells (HSPCs) to treat severe inherited disorders or cancer, have set the stage for the ... ...

Abstract	Lessons learned from decades-long practice in the transplantation of hematopoietic stem and progenitor cells (HSPCs) to treat severe inherited disorders or cancer, have set the stage for the current
Language	English
Publishing date	2023-01-09
Publishing country	Switzerland
Document type	Journal Article ; Review
ISSN	2673-3439
ISSN (online)	2673-3439
DOI	10.3389/fgeed.2022.997142
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Article ; Online: Recent Advances Using Genetic Therapies Against Infectious Diseases and for Vaccination.

Galy, Anne / Berkhout, Ben / Breckpot, Karine / Pichon, Chantal / Bloom, Kristie / Kiem, Hans-Peter / Mühlebach, Michael D / McCune, Joseph M

Human gene therapy

2023 Volume 34, Issue 17-18, Page(s) 896–904

Abstract: The development of prophylatic or therapeutic medicines for infectious diseases is one of the priorities for health organizations worldwide. Innovative solutions are required to achieve effective, safe, and accessible treatments for most if not all ... ...

Abstract	The development of prophylatic or therapeutic medicines for infectious diseases is one of the priorities for health organizations worldwide. Innovative solutions are required to achieve effective, safe, and accessible treatments for most if not all infectious diseases, particularly those that are chronic in nature or that emerge unexpectedly over time. Genetic technologies offer versatile possibilities to design therapies against pathogens. Recent developments such as mRNA vaccines, CRISPR gene editing, and immunotherapies provide unprecedented hope to achieve significant results in the field of infectious diseases. This review will focus on advances in this domain, showcasing the cross-fertilization with other fields (
MeSH term(s)	Humans ; Communicable Diseases/genetics ; Communicable Diseases/therapy ; Genetic Therapy ; Vaccination ; Cloning, Molecular ; Clustered Regularly Interspaced Short Palindromic Repeats
Language	English
Publishing date	2023-08-28
Publishing country	United States
Document type	Journal Article ; Review ; Research Support, Non-U.S. Gov't
ZDB-ID	1028152-6
ISSN	1557-7422 ; 1043-0342
ISSN (online)	1557-7422
ISSN	1043-0342
DOI	10.1089/hum.2023.123
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Zs.A 2988: Show issues

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Article ; Online: Haemolytic paroxysmal nocturnal haemoglobinuria in patients with myeloid neoplasms: A rare association with specific therapeutic implications.

Sutra Del Galy, Aurélien / Willems, Lise / D'Aveni, Maud / Pautas, Cécile / Chantepie, Sylvain / Carpentier, Benjamin / Barraco, Fiorenza / Banos, Anne / Garidi, Reda / Forcade, Edouard / Sicre de Fontbrune, Flore / Peffault de Latour, Régis

British journal of haematology

2023 Volume 201, Issue 2, Page(s) e16–e20

MeSH term(s)	Humans ; Hemoglobinuria, Paroxysmal/therapy ; Hemoglobinuria, Paroxysmal/drug therapy ; Neoplasms/complications ; Myeloproliferative Disorders/complications ; Hemolysis
Language	English
Publishing date	2023-02-17
Publishing country	England
Document type	Letter
ZDB-ID	80077-6
ISSN	1365-2141 ; 0007-1048
ISSN (online)	1365-2141
ISSN	0007-1048
DOI	10.1111/bjh.18693
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Article ; Online: An empowered, clinically viable hematopoietic stem cell gene therapy for the treatment of multisystemic mucopolysaccharidosis type II.

Das, Sabyasachi / Rruga, Fatlum / Montepeloso, Annita / Dimartino, Agnese / Spadini, Silvia / Corre, Guillaume / Patel, Janki / Cavalca, Eleonora / Ferro, Francesca / Gatti, Alessandra / Milazzo, Rita / Galy, Anne / Politi, Letterio S / Rizzardi, Gian Paolo / Vallanti, Giuliana / Poletti, Valentina / Biffi, Alessandra

Molecular therapy : the journal of the American Society of Gene Therapy

2024 Volume 32, Issue 3, Page(s) 619–636

Abstract: Mucopolysaccharidosis type II (MPS II), or Hunter syndrome, is a rare X-linked recessive lysosomal storage disorder due to a mutation in the lysosomal enzyme iduronate-2-sulfatase (IDS) gene. IDS deficiency leads to a progressive, multisystem ... ...

Abstract	Mucopolysaccharidosis type II (MPS II), or Hunter syndrome, is a rare X-linked recessive lysosomal storage disorder due to a mutation in the lysosomal enzyme iduronate-2-sulfatase (IDS) gene. IDS deficiency leads to a progressive, multisystem accumulation of glycosaminoglycans (GAGs) and results in central nervous system (CNS) manifestations in the severe form. We developed up to clinical readiness a new hematopoietic stem cell (HSC) gene therapy approach for MPS II that benefits from a novel highly effective transduction protocol. We first provided proof of concept of efficacy of our approach aimed at enhanced IDS enzyme delivery to the CNS in a murine study of immediate translational value, employing a lentiviral vector (LV) encoding a codon-optimized human IDS cDNA. Then the therapeutic LV was tested for its ability to efficiently and safely transduce bona fide human HSCs in clinically relevant conditions according to a standard vs. a novel protocol that demonstrated superior ability to transduce bona fide long-term repopulating HSCs. Overall, these results provide strong proof of concept for the clinical translation of this approach for the treatment of Hunter syndrome.
MeSH term(s)	Humans ; Animals ; Mice ; Mucopolysaccharidosis II/therapy ; Mucopolysaccharidosis II/drug therapy ; Iduronate Sulfatase/genetics ; Iduronate Sulfatase/metabolism ; Genetic Therapy ; Central Nervous System/metabolism ; Lentivirus/genetics ; Lentivirus/metabolism ; Hematopoietic Stem Cells/metabolism
Chemical Substances	Iduronate Sulfatase (EC 3.1.6.13)
Language	English
Publishing date	2024-02-03
Publishing country	United States
Document type	Journal Article
ZDB-ID	2010592-7
ISSN	1525-0024 ; 1525-0016
ISSN (online)	1525-0024
ISSN	1525-0016
DOI	10.1016/j.ymthe.2024.01.034
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Article ; Online: Lentiviral standards to determine the sensitivity of assays that quantify lentiviral vector copy numbers and genomic insertion sites in cells.

Corre, Guillaume / Seye, Ababacar / Frin, Sophie / Ferrand, Maxime / Winkler, Kathrin / Luc, Cyril / Dorange, Fabien / Rocca, Céline J / Galy, Anne

Gene therapy

2022 Volume 29, Issue 9, Page(s) 536–543

Abstract: With an increasing number of gene therapy clinical trials and drugs reaching the market, it becomes important to standardize the methods that evaluate the efficacy and safety of gene therapy. We herein report the generation of lentiviral standards which ... ...

Abstract	With an increasing number of gene therapy clinical trials and drugs reaching the market, it becomes important to standardize the methods that evaluate the efficacy and safety of gene therapy. We herein report the generation of lentiviral standards which are stable, cloned human cells prepared from the diploid HCT116 cell line and which carry a known number of lentiviral vector copies in their genome. These clones can be used as reference cellular materials for the calibration or qualification of analytical methods that quantify vector copy numbers in cells (VCN) or lentiviral vector genomic integration sites (IS). Cellular standards were used to show the superior precision of digital droplet PCR (ddPCR) over quantitative PCR (qPCR) for VCN determination. This enabled us to develop a new sensitive and specific VCN ddPCR method specific for the integrated provirus and not recognizing the transfer plasmid. The cellular standards, were also useful to assess the sensitivity and limits of a ligation-mediated PCR (LM-PCR) method to measure IS showing that at least 1% abundance of a single IS can be detected in a polyclonal population but that not all IS can be amplified with similar efficiency. Thus, lentiviral standards should be systematically used in all assays that assess lentiviral gene therapy efficacy and safety.
MeSH term(s)	DNA Copy Number Variations ; Genetic Therapy ; Genomics ; Humans ; Real-Time Polymerase Chain Reaction
Language	English
Publishing date	2022-02-22
Publishing country	England
Document type	Journal Article ; Research Support, Non-U.S. Gov't
ZDB-ID	1191036-7
ISSN	1476-5462 ; 0969-7128
ISSN (online)	1476-5462
ISSN	0969-7128
DOI	10.1038/s41434-022-00315-8
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