Pub Date : 2023-11-20DOI: 10.1038/s41434-023-00428-8
A. Mateu-Bosch, E. Segur-Bailach, J. García-Villoria, S. Gea-Sorlí, I. Ruiz, J. del Rey, J. Camps, M. Guitart-Mampel, G. Garrabou, F. Tort, A. Ribes, C. Fillat
Glutaric Aciduria type I (GA1) is a rare neurometabolic disorder caused by mutations in the GDCH gene encoding for glutaryl-CoA dehydrogenase (GCDH) in the catabolic pathway of lysine, hydroxylysine and tryptophan. GCDH deficiency leads to increased concentrations of glutaric acid (GA) and 3-hydroxyglutaric acid (3-OHGA) in body fluids and tissues. These metabolites are the main triggers of brain damage. Mechanistic studies supporting neurotoxicity in mouse models have been conducted. However, the different vulnerability to some stressors between mouse and human brain cells reveals the need to have a reliable human neuronal model to study GA1 pathogenesis. In the present work we generated a GCDH knockout (KO) in the human neuroblastoma cell line SH-SY5Y by CRISPR/Cas9 technology. SH-SY5Y-GCDH KO cells accumulate GA, 3-OHGA, and glutarylcarnitine when exposed to lysine overload. GA or lysine treatment triggered neuronal damage in GCDH deficient cells. SH-SY5Y-GCDH KO cells also displayed features of GA1 pathogenesis such as increased oxidative stress vulnerability. Restoration of the GCDH activity by gene replacement rescued neuronal alterations. Thus, our findings provide a human neuronal cellular model of GA1 to study this disease and show the potential of gene therapy to rescue GCDH deficiency.
{"title":"Modeling Glutaric Aciduria Type I in human neuroblastoma cells recapitulates neuronal damage that can be rescued by gene replacement","authors":"A. Mateu-Bosch, E. Segur-Bailach, J. García-Villoria, S. Gea-Sorlí, I. Ruiz, J. del Rey, J. Camps, M. Guitart-Mampel, G. Garrabou, F. Tort, A. Ribes, C. Fillat","doi":"10.1038/s41434-023-00428-8","DOIUrl":"10.1038/s41434-023-00428-8","url":null,"abstract":"Glutaric Aciduria type I (GA1) is a rare neurometabolic disorder caused by mutations in the GDCH gene encoding for glutaryl-CoA dehydrogenase (GCDH) in the catabolic pathway of lysine, hydroxylysine and tryptophan. GCDH deficiency leads to increased concentrations of glutaric acid (GA) and 3-hydroxyglutaric acid (3-OHGA) in body fluids and tissues. These metabolites are the main triggers of brain damage. Mechanistic studies supporting neurotoxicity in mouse models have been conducted. However, the different vulnerability to some stressors between mouse and human brain cells reveals the need to have a reliable human neuronal model to study GA1 pathogenesis. In the present work we generated a GCDH knockout (KO) in the human neuroblastoma cell line SH-SY5Y by CRISPR/Cas9 technology. SH-SY5Y-GCDH KO cells accumulate GA, 3-OHGA, and glutarylcarnitine when exposed to lysine overload. GA or lysine treatment triggered neuronal damage in GCDH deficient cells. SH-SY5Y-GCDH KO cells also displayed features of GA1 pathogenesis such as increased oxidative stress vulnerability. Restoration of the GCDH activity by gene replacement rescued neuronal alterations. Thus, our findings provide a human neuronal cellular model of GA1 to study this disease and show the potential of gene therapy to rescue GCDH deficiency.","PeriodicalId":12699,"journal":{"name":"Gene Therapy","volume":"31 1-2","pages":"12-18"},"PeriodicalIF":5.1,"publicationDate":"2023-11-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138176074","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2023-11-15DOI: 10.1038/s41434-023-00427-9
Evgeny S. Nikitin, Tatiana Y. Postnikova, Elena Y. Proskurina, Anastasia A. Borodinova, Violetta Ivanova, Matvey V. Roshchin, Maria P. Smirnova, Ilya Kelmanson, Vsevolod V. Belousov, Pavel M. Balaban, Aleksey V. Zaitsev
Gene therapy offers a potential alternative to the surgical treatment of epilepsy, which affects millions of people and is pharmacoresistant in ~30% of cases. Aimed at reducing the excitability of principal neurons, the engineered expression of K+ channels has been proposed as a treatment due to the outstanding ability of K+ channels to hyperpolarize neurons. However, the effects of K+ channel overexpression on cell physiology remain to be investigated. Here we report an adeno-associated virus (AAV) vector designed to reduce epileptiform activity specifically in excitatory pyramidal neurons by expressing the human Ca2+-gated K+ channel KCNN4 (KCa3.1). Electrophysiological and pharmacological experiments in acute brain slices showed that KCNN4-transduced cells exhibited a Ca2+-dependent slow afterhyperpolarization that significantly decreased the ability of KCNN4-positive neurons to generate high-frequency spike trains without affecting their lower-frequency coding ability and action potential shapes. Antiepileptic activity tests showed potent suppression of pharmacologically induced seizures in vitro at both single cell and local field potential levels with decreased spiking during ictal discharges. Taken together, our findings strongly suggest that the AAV-based expression of the KCNN4 channel in excitatory neurons is a promising therapeutic intervention as gene therapy for epilepsy.
{"title":"Overexpression of KCNN4 channels in principal neurons produces an anti-seizure effect without reducing their coding ability","authors":"Evgeny S. Nikitin, Tatiana Y. Postnikova, Elena Y. Proskurina, Anastasia A. Borodinova, Violetta Ivanova, Matvey V. Roshchin, Maria P. Smirnova, Ilya Kelmanson, Vsevolod V. Belousov, Pavel M. Balaban, Aleksey V. Zaitsev","doi":"10.1038/s41434-023-00427-9","DOIUrl":"10.1038/s41434-023-00427-9","url":null,"abstract":"Gene therapy offers a potential alternative to the surgical treatment of epilepsy, which affects millions of people and is pharmacoresistant in ~30% of cases. Aimed at reducing the excitability of principal neurons, the engineered expression of K+ channels has been proposed as a treatment due to the outstanding ability of K+ channels to hyperpolarize neurons. However, the effects of K+ channel overexpression on cell physiology remain to be investigated. Here we report an adeno-associated virus (AAV) vector designed to reduce epileptiform activity specifically in excitatory pyramidal neurons by expressing the human Ca2+-gated K+ channel KCNN4 (KCa3.1). Electrophysiological and pharmacological experiments in acute brain slices showed that KCNN4-transduced cells exhibited a Ca2+-dependent slow afterhyperpolarization that significantly decreased the ability of KCNN4-positive neurons to generate high-frequency spike trains without affecting their lower-frequency coding ability and action potential shapes. Antiepileptic activity tests showed potent suppression of pharmacologically induced seizures in vitro at both single cell and local field potential levels with decreased spiking during ictal discharges. Taken together, our findings strongly suggest that the AAV-based expression of the KCNN4 channel in excitatory neurons is a promising therapeutic intervention as gene therapy for epilepsy.","PeriodicalId":12699,"journal":{"name":"Gene Therapy","volume":"31 3-4","pages":"144-153"},"PeriodicalIF":5.1,"publicationDate":"2023-11-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"134648784","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2023-11-08DOI: 10.1038/s41434-023-00390-5
Donald B. Kohn, Yvonne Y. Chen, Melissa J. Spencer
Despite the ups and downs in the field over three decades, the science of gene therapy has continued to advance and provide enduring treatments for increasing number of diseases. There are active clinical trials approaching a variety of inherited and acquired disorders of different organ systems. Approaches include ex vivo modification of hematologic stem cells (HSC), T lymphocytes and other immune cells, as well as in vivo delivery of genes or gene editing reagents to the relevant target cells by either local or systemic administration. In this article, we highlight success and ongoing challenges in three areas of high activity in gene therapy: inherited blood cell diseases by targeting hematopoietic stem cells, malignant disorders using immune effector cells genetically modified with chimeric antigen receptors, and ophthalmologic, neurologic, and coagulation disorders using in vivo administration of adeno-associated virus (AAV) vectors. In recent years, there have been true cures for many of these diseases, with sustained clinical benefit that exceed those from other medical approaches. Each of these treatments faces ongoing challenges, namely their high one-time costs and the complexity of manufacturing the therapeutic agents, which are biological viruses and cell products, at pharmacologic standards of quality and consistency. New models of reimbursement are needed to make these innovative treatments widely available to patients in need.
{"title":"Successes and challenges in clinical gene therapy","authors":"Donald B. Kohn, Yvonne Y. Chen, Melissa J. Spencer","doi":"10.1038/s41434-023-00390-5","DOIUrl":"10.1038/s41434-023-00390-5","url":null,"abstract":"Despite the ups and downs in the field over three decades, the science of gene therapy has continued to advance and provide enduring treatments for increasing number of diseases. There are active clinical trials approaching a variety of inherited and acquired disorders of different organ systems. Approaches include ex vivo modification of hematologic stem cells (HSC), T lymphocytes and other immune cells, as well as in vivo delivery of genes or gene editing reagents to the relevant target cells by either local or systemic administration. In this article, we highlight success and ongoing challenges in three areas of high activity in gene therapy: inherited blood cell diseases by targeting hematopoietic stem cells, malignant disorders using immune effector cells genetically modified with chimeric antigen receptors, and ophthalmologic, neurologic, and coagulation disorders using in vivo administration of adeno-associated virus (AAV) vectors. In recent years, there have been true cures for many of these diseases, with sustained clinical benefit that exceed those from other medical approaches. Each of these treatments faces ongoing challenges, namely their high one-time costs and the complexity of manufacturing the therapeutic agents, which are biological viruses and cell products, at pharmacologic standards of quality and consistency. New models of reimbursement are needed to make these innovative treatments widely available to patients in need.","PeriodicalId":12699,"journal":{"name":"Gene Therapy","volume":"30 10-11","pages":"738-746"},"PeriodicalIF":5.1,"publicationDate":"2023-11-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10678346/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"71480727","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2023-11-08DOI: 10.1038/s41434-023-00393-2
Steven Joffe, Rena M. Conti, Jorge L. Contreras, Emily A. Largent, Holly Fernandez Lynch, David Mitchell, Rachel E. Sachs, Allison M. Whelan, Matthew S. McCoy
{"title":"Access to affordable medicines: obligations of universities and academic medical centers","authors":"Steven Joffe, Rena M. Conti, Jorge L. Contreras, Emily A. Largent, Holly Fernandez Lynch, David Mitchell, Rachel E. Sachs, Allison M. Whelan, Matthew S. McCoy","doi":"10.1038/s41434-023-00393-2","DOIUrl":"10.1038/s41434-023-00393-2","url":null,"abstract":"","PeriodicalId":12699,"journal":{"name":"Gene Therapy","volume":"30 10-11","pages":"753-755"},"PeriodicalIF":5.1,"publicationDate":"2023-11-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"71480724","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2023-11-08DOI: 10.1038/s41434-023-00402-4
Kerstin N. Vokinger, Camille E. G. Glaus, Aaron S. Kesselheim
Gene therapies are a fast-growing area of innovation and hold promise for the treatment of many diseases currently with unmet medical need. To better understand the clinical importance of the current landscape of approved gene therapies, we conducted a systematic analysis of the approved gene therapies and their added therapeutic value. Through December 2022, 13 gene therapies have been approved in the US, 15 in the EU, and 9 in Switzerland. Nine gene therapies have been approved in all three jurisdictions, and 11 in both the US and EU. Among the 11 gene therapies approved in more than one jurisdiction, there were differences in the approved indications among the regulatory agencies, mostly the European drug agencies (EMA and Swissmedic) being more restrictive. Among the gene therapies with available therapeutic ratings, approximately two-thirds had high added therapeutic value, which is substantially higher than the average prevalence of high added therapeutic value ratings among new drugs and biologics (approximately one-third). However, therapies with high added therapeutic value will not be useful for patients if excessive prices limit access to them. Drug pricing reforms should address gene therapies to ensure access to new gene therapies that can offer important therapeutic value to patients.
{"title":"Approval and therapeutic value of gene therapies in the US and Europe","authors":"Kerstin N. Vokinger, Camille E. G. Glaus, Aaron S. Kesselheim","doi":"10.1038/s41434-023-00402-4","DOIUrl":"10.1038/s41434-023-00402-4","url":null,"abstract":"Gene therapies are a fast-growing area of innovation and hold promise for the treatment of many diseases currently with unmet medical need. To better understand the clinical importance of the current landscape of approved gene therapies, we conducted a systematic analysis of the approved gene therapies and their added therapeutic value. Through December 2022, 13 gene therapies have been approved in the US, 15 in the EU, and 9 in Switzerland. Nine gene therapies have been approved in all three jurisdictions, and 11 in both the US and EU. Among the 11 gene therapies approved in more than one jurisdiction, there were differences in the approved indications among the regulatory agencies, mostly the European drug agencies (EMA and Swissmedic) being more restrictive. Among the gene therapies with available therapeutic ratings, approximately two-thirds had high added therapeutic value, which is substantially higher than the average prevalence of high added therapeutic value ratings among new drugs and biologics (approximately one-third). However, therapies with high added therapeutic value will not be useful for patients if excessive prices limit access to them. Drug pricing reforms should address gene therapies to ensure access to new gene therapies that can offer important therapeutic value to patients.","PeriodicalId":12699,"journal":{"name":"Gene Therapy","volume":"30 10-11","pages":"756-760"},"PeriodicalIF":5.1,"publicationDate":"2023-11-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"71480725","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2023-11-08DOI: 10.1038/s41434-023-00392-3
Lea Witkowsky, Matthew Norstad, Audrey R. Glynn, Melinda Kliegman
{"title":"Towards affordable CRISPR genomic therapies: a task force convened by the Innovative Genomics Institute","authors":"Lea Witkowsky, Matthew Norstad, Audrey R. Glynn, Melinda Kliegman","doi":"10.1038/s41434-023-00392-3","DOIUrl":"10.1038/s41434-023-00392-3","url":null,"abstract":"","PeriodicalId":12699,"journal":{"name":"Gene Therapy","volume":"30 10-11","pages":"747-752"},"PeriodicalIF":5.1,"publicationDate":"2023-11-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10678297/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"71480729","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2023-11-08DOI: 10.1038/s41434-023-00419-9
Chi Heem Wong, Dexin Li, Nina Wang, Jonathan Gruber, Andrew W. Lo, Rena M. Conti
Gene therapy is a new class of medical treatment that alters part of a patient’s genome through the replacement, deletion, or insertion of genetic material. While still in its infancy, gene therapy has demonstrated immense potential to treat and even cure previously intractable diseases. Nevertheless, existing gene therapy prices are high, raising concerns about its affordability for U.S. payers and its availability to patients. We assess the potential financial impact of novel gene therapies by developing and implementing an original simulation model which entails the following steps: identifying the 109 late-stage gene therapy clinical trials underway before January 2020, estimating the prevalence and incidence of their corresponding diseases, applying a model of the increase in quality-adjusted life years for each therapy, and simulating the launch prices and expected spending of all available gene therapies annually. The results of our simulation suggest that annual spending on gene therapies will be approximately $20.4 billion, under conservative assumptions. We decompose the estimated spending by treated age group as a proxy for insurance type, finding that approximately one-half of annual spending will on the use of gene therapies to treat non-Medicare-insured adults and children. We conduct multiple sensitivity analyses regarding our assumptions and model parameters. We conclude by considering the tradeoffs of different payment methods and policies that intend to ensure patient access to the expected benefits of gene therapy.
{"title":"The estimated annual financial impact of gene therapy in the United States","authors":"Chi Heem Wong, Dexin Li, Nina Wang, Jonathan Gruber, Andrew W. Lo, Rena M. Conti","doi":"10.1038/s41434-023-00419-9","DOIUrl":"10.1038/s41434-023-00419-9","url":null,"abstract":"Gene therapy is a new class of medical treatment that alters part of a patient’s genome through the replacement, deletion, or insertion of genetic material. While still in its infancy, gene therapy has demonstrated immense potential to treat and even cure previously intractable diseases. Nevertheless, existing gene therapy prices are high, raising concerns about its affordability for U.S. payers and its availability to patients. We assess the potential financial impact of novel gene therapies by developing and implementing an original simulation model which entails the following steps: identifying the 109 late-stage gene therapy clinical trials underway before January 2020, estimating the prevalence and incidence of their corresponding diseases, applying a model of the increase in quality-adjusted life years for each therapy, and simulating the launch prices and expected spending of all available gene therapies annually. The results of our simulation suggest that annual spending on gene therapies will be approximately $20.4 billion, under conservative assumptions. We decompose the estimated spending by treated age group as a proxy for insurance type, finding that approximately one-half of annual spending will on the use of gene therapies to treat non-Medicare-insured adults and children. We conduct multiple sensitivity analyses regarding our assumptions and model parameters. We conclude by considering the tradeoffs of different payment methods and policies that intend to ensure patient access to the expected benefits of gene therapy.","PeriodicalId":12699,"journal":{"name":"Gene Therapy","volume":"30 10-11","pages":"761-773"},"PeriodicalIF":5.1,"publicationDate":"2023-11-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10678302/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"71480728","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2023-10-30DOI: 10.1038/s41434-023-00425-x
Shadi Saleh, Omar Dabbous, Sean D. Sullivan, Dipen Ankleshwaria, Daiane Trombini, Mondher Toumi, Mahmoud Diaa, Anish Patel, Burcu Kazazoglu Taylor, Sean Tunis
In the rapidly evolving landscape of biotechnologies, cell and gene therapies are being developed and adopted at an unprecedented pace. However, their access and adoption remain limited, particularly in low- and middle-income countries (LMICs). This study aims to address this critical gap by exploring the potential of applying a hub and spoke model for cell and gene therapy delivery in LMICs. We establish the identity and roles of relevant stakeholders, propose a hub and spoke model for cell and gene therapy delivery, and simulate its application in Brazil and the Middle East and North Africa. The development and simulation of this model were informed by a comprehensive review of academic articles, grey literature, relevant websites, and publicly available data sets. The proposed hub and spoke model is expected to expand availability of and access to cell and gene therapy in LMICs and presents a comprehensive framework for the roles of core stakeholders, laying the groundwork for more equitable access to these lifesaving therapies. More research is needed to explore the practical adoption and implications of this model.
{"title":"A practical approach for adoption of a hub and spoke model for cell and gene therapies in low- and middle-income countries: framework and case studies","authors":"Shadi Saleh, Omar Dabbous, Sean D. Sullivan, Dipen Ankleshwaria, Daiane Trombini, Mondher Toumi, Mahmoud Diaa, Anish Patel, Burcu Kazazoglu Taylor, Sean Tunis","doi":"10.1038/s41434-023-00425-x","DOIUrl":"10.1038/s41434-023-00425-x","url":null,"abstract":"In the rapidly evolving landscape of biotechnologies, cell and gene therapies are being developed and adopted at an unprecedented pace. However, their access and adoption remain limited, particularly in low- and middle-income countries (LMICs). This study aims to address this critical gap by exploring the potential of applying a hub and spoke model for cell and gene therapy delivery in LMICs. We establish the identity and roles of relevant stakeholders, propose a hub and spoke model for cell and gene therapy delivery, and simulate its application in Brazil and the Middle East and North Africa. The development and simulation of this model were informed by a comprehensive review of academic articles, grey literature, relevant websites, and publicly available data sets. The proposed hub and spoke model is expected to expand availability of and access to cell and gene therapy in LMICs and presents a comprehensive framework for the roles of core stakeholders, laying the groundwork for more equitable access to these lifesaving therapies. More research is needed to explore the practical adoption and implications of this model.","PeriodicalId":12699,"journal":{"name":"Gene Therapy","volume":"31 1-2","pages":"1-11"},"PeriodicalIF":5.1,"publicationDate":"2023-10-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.nature.com/articles/s41434-023-00425-x.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"71411948","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2023-10-26DOI: 10.1038/s41434-023-00426-w
Dandan Zhu, Mehri Barabadi, Courtney McDonald, Gina Kusuma, Ishmael Miguel Inocencio, Rebecca Lim
Cell based therapies are being assessed for their therapeutic potential across a variety of diseases. Gestational tissues are attractive sources for cell therapy. The large number of births worldwide ensures sufficient access to gestational tissues, however, limited information has been reported around the impact of birth trends, delivery methods and pregnancy conditions on perinatal stem cell banking. This review describes the current state of banking of gestational tissues and their derived perinatal stem cells, discusses why the changes in birth trends and delivery methods could affect gestational tissue banking practices, and further explores how common pregnancy complications can potentially influence perinatal stem cell banking.
{"title":"Implications of maternal-fetal health on perinatal stem cell banking","authors":"Dandan Zhu, Mehri Barabadi, Courtney McDonald, Gina Kusuma, Ishmael Miguel Inocencio, Rebecca Lim","doi":"10.1038/s41434-023-00426-w","DOIUrl":"10.1038/s41434-023-00426-w","url":null,"abstract":"Cell based therapies are being assessed for their therapeutic potential across a variety of diseases. Gestational tissues are attractive sources for cell therapy. The large number of births worldwide ensures sufficient access to gestational tissues, however, limited information has been reported around the impact of birth trends, delivery methods and pregnancy conditions on perinatal stem cell banking. This review describes the current state of banking of gestational tissues and their derived perinatal stem cells, discusses why the changes in birth trends and delivery methods could affect gestational tissue banking practices, and further explores how common pregnancy complications can potentially influence perinatal stem cell banking.","PeriodicalId":12699,"journal":{"name":"Gene Therapy","volume":"31 3-4","pages":"65-73"},"PeriodicalIF":5.1,"publicationDate":"2023-10-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.nature.com/articles/s41434-023-00426-w.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"50161457","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
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