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1.
Nihon Yakurigaku Zasshi ; 157(1): 53-61, 2022.
Artigo em Japonês | MEDLINE | ID: mdl-34980814

RESUMO

Onasemnogene abeparvovec (Zolgensma®; formerly AVXS-101) is a one-time gene therapy designed to address the genetic root cause of spinal muscular atrophy (SMA) by replacing the function of the missing or nonworking SMN1 gene via an adeno-associated AAV9 viral vector. On March 19, 2020, the Japanese Ministry of Health, Labor and Welfare approved onasemnogene abeparvovec for the treatment of SMA patients <2 years of age, including presymptomatic patients with a genetic diagnosis. Patients must be negative for elevated anti-AAV9 antibodies. Onasemnogene abeparvovec is administered through a single intravenous infusion, delivering a new working copy of the SMN gene into a patient's cells. Intravenous administration of onasemnogene abeparvovec to SMA model mice resulted in sustained expression of survival motor neuron (SMN) protein, weight gain, improvement of motor function, and prolongation of survival. Its clinical efficacy and safety have been demonstrated through the Phase I START and Phase III STR1VE-US, STR1VE-EU, and SPR1NT trials, and their long-term extension studies. SMA and presymptomatic patients treated with onasemnogene abeparvovec have achieved rates of survival not observed in the natural history of SMA. Treatment has led to rapid motor function improvement, often within one month of dosing, and developmental milestone achievement, including the ability to sit without support. The most commonly observed adverse effects after treatment were elevated liver enzymes, which often resolved with a course of prednisolone, and vomiting. This review discusses the rationale underlying gene replacement therapy for SMA, and describes the basic science, clinical trial experience, and use of onasemnogene abeparvovec.


Assuntos
Atrofia Muscular Espinal , Atrofias Musculares Espinais da Infância , Animais , Terapia Genética , Humanos , Infusões Intravenosas , Camundongos , Atrofia Muscular Espinal/genética , Atrofia Muscular Espinal/terapia , Atrofias Musculares Espinais da Infância/tratamento farmacológico , Resultado do Tratamento
2.
Biomed Pharmacother ; 145: 112385, 2022 Jan.
Artigo em Inglês | MEDLINE | ID: mdl-34915673

RESUMO

Chemically modified mRNA represents a unique, efficient, and straightforward approach to produce a class of biopharmaceutical agents. It has been already approved as a vaccination-based method for targeting SARS-CoV-2 virus. The COVID-19 pandemic has highlighted the prospect of synthetic modified mRNA to efficiently and safely combat various diseases. Recently, various optimization advances have been adopted to overcome the limitations associated with conventional gene therapeutics leading to wide-ranging applications in different disease conditions. This review sheds light on emerging directions of chemically modified mRNAs to prevent and treat widespread chronic diseases, including metabolic disorders, cancer vaccination and immunotherapy, musculoskeletal disorders, respiratory conditions, cardiovascular diseases, and liver diseases.


Assuntos
COVID-19/prevenção & controle , Doença Crônica/prevenção & controle , Doença Crônica/terapia , Terapia Genética/métodos , Imunoterapia/métodos , Pandemias/prevenção & controle , RNA Mensageiro/química , SARS-CoV-2/imunologia , Vacinas Sintéticas , Disponibilidade Biológica , Portadores de Fármacos , Previsões , Técnicas de Transferência de Genes , Vetores Genéticos/administração & dosagem , Vetores Genéticos/uso terapêutico , Humanos , Imunoterapia Ativa , Estabilidade de RNA , RNA Mensageiro/administração & dosagem , RNA Mensageiro/imunologia , RNA Mensageiro/uso terapêutico , SARS-CoV-2/genética , Vacinas Sintéticas/administração & dosagem , Vacinas Sintéticas/imunologia , /imunologia
5.
Cell Biochem Funct ; 40(1): 28-48, 2022 Jan.
Artigo em Inglês | MEDLINE | ID: mdl-34904722

RESUMO

Breast cancer (BC) is the most prevalent malignancy and the second leading cause of death among women worldwide that is caused by numerous genetic and environmental factors. Hence, effective treatment for this type of cancer requires new therapeutic approaches. The traditional methods for treating this cancer have side effects, therefore so much research have been performed in last decade to find new methods to alleviate these problems. The study of the molecular basis of breast cancer has led to the introduction of gene therapy as an effective therapeutic approach for this cancer. Gene therapy involves sending genetic material through a vector into target cells, which is followed by a correction, addition, or suppression of the gene. In this technique, it is necessary to target tumour cells without affecting normal cells. In addition, clinical trial studies have shown that this approach is less toxic than traditional therapies. This study will review various aspects of breast cancer, gene therapy strategies, limitations, challenges and recent studies in this area.


Assuntos
Neoplasias da Mama , Neoplasias da Mama/genética , Neoplasias da Mama/terapia , Feminino , Terapia Genética , Humanos
6.
World Neurosurg ; 157: 282-299, 2022 01.
Artigo em Inglês | MEDLINE | ID: mdl-34929786

RESUMO

Low back pain is the leading cause of work absences and years lived with disability, and it is often associated with degenerative disc disease. In recent years, biological treatment approaches such as the use of growth factors, cell injections, annulus fibrosus (AF) repair, nucleus pulposus replacement, and tissue-engineered discs have been explored as means for preventing or reversing degenerative disc disease. Both animal and clinical studies have shown promising results for cell-based therapy on the grounds of its regenerative potential. Clinical data also indicate that stem cell injection is safe when appropriately performed, albeit its long-term safety and efficacy are yet to be explored. Numerous challenges also remain to be overcome, such as isolating, differentiating, and preconditioning the disc cells, as well as managing the nutrient-deficient and oxygen-deficient micromilieu of the intervertebral disc (IVD). AF repair methods including devices used in clinical trials have shown success in decreasing reherniation rates and improving overall clinical outcomes. In addition, recent studies that combined AF repair and nucleus pulposus replacement have shown improved biomechanical stability in IVDs after the combined treatment. Tissue-engineered IVDs for total disc replacement are still being developed, and future studies are necessary to overcome the challenges in their delivery, efficacy, and safety.


Assuntos
Produtos Biológicos/uso terapêutico , Fenômenos Biomecânicos/fisiologia , Degeneração do Disco Intervertebral/fisiopatologia , Degeneração do Disco Intervertebral/terapia , Terapias em Estudo/métodos , Animais , Produtos Biológicos/farmacologia , Fenômenos Biomecânicos/efeitos dos fármacos , Ensaios Clínicos como Assunto/métodos , Terapia Genética/métodos , Terapia Genética/tendências , Humanos , Degeneração do Disco Intervertebral/diagnóstico , Terapias em Estudo/tendências , Engenharia Tecidual/métodos , Engenharia Tecidual/tendências , Substituição Total de Disco/métodos , Substituição Total de Disco/tendências , Resultado do Tratamento
7.
J Manag Care Spec Pharm ; 28(1): 39-47, 2022 Jan.
Artigo em Inglês | MEDLINE | ID: mdl-34949120

RESUMO

BACKGROUND: Genetic therapies are a promising treatment for children born with spinal muscular atrophy (SMA); however, their high price tags can evoke coverage restrictions. OBJECTIVE: To assess variation in coverage guidelines across fee-for-service state Medicaid programs for 2 novel genetic therapies, nusinersen and onasemnogene abeparvovec, that treat SMA. We also assessed the association of these coverage guidelines with use of the 2 genetic therapies. METHODS: We evaluated fee-for-service Medicaid coverage policies for nusinersen and onasemnogene abeparvovec from publicly available websites for the period February 2020-March 2020. We then documented areas of agreement and disagreement across 4 key coverage domains. We used 2018 and 2019 state Medicaid drug utilization data to calculate the use of nusinersen across Medicaid programs and assessed that use against the restrictiveness of the coverage guidelines. RESULTS: We identified 19 state Medicaid coverage guidelines for nusinersen. Most states agreed on diagnostics requirements; however, there were disagreements based on ventilator status. We identified 17 state Medicaid coverage guidelines for onasemnogene abeparvovec. There was more discordance in these coverage guidelines compared with nusinersen, notably in domains of SMN2 gene count and ventilator status. When comparing utilization of nusinersen with coverage restrictions, we found that the more restrictive states had considerably lower utilization of nusinersen. CONCLUSIONS: There was significant variation across fee-for-service Medicaid coverage policies for nusinersen and onasemnogene abeparvovec. Although states can impose individual coverage guidelines for each drug, we presented policy options that could reduce variation and potentially decrease the cost burden of these drugs. DISCLOSURES: This study was funded by Arnold Ventures. The authors have no conflicts of interest to disclose.


Assuntos
Terapia Genética , Cobertura do Seguro/estatística & dados numéricos , Medicaid , Atrofia Muscular Espinal/tratamento farmacológico , Terapia Genética/economia , Humanos , Estados Unidos
8.
J Biomed Nanotechnol ; 17(11): 2114-2124, 2021 Nov 01.
Artigo em Inglês | MEDLINE | ID: mdl-34906273

RESUMO

The nanometer size and biological characteristics of recombinant adeno-associated virus vectors (rAAV) make them particularly useful as gene therapy vectors and they have been successfully used in this role. Our latest research revealed that the rAAV/DJ/CAG mosaic vector offers highly efficient targeted gene delivery to melanoma cells metastasized to the lungs and that the transduction is temperature dependent. In order to further explore the ability of the rAAV/DJ/CAG vector to deliver highly selective transduction, this study was designed to identify the transduction stability of rAAV/DJ/CAG under various conditions. The temperatures used in this study ranged from -196 ° (liquid nitrogen) to 90 °, and the effect of temperature fluctuations (freeze-thaw, cooling-heating cycles) was also studied. This research also investigated the effects of UV radiation (ultraviolet) on the rAAV/DJ/CAG activity. Changes in the transduction efficiency were assessed via fluorescence microscopy imaging and the qPCR method. Under the test conditions, the transduction efficiency was reduced by approx. 35%, on average. High temperatures (70 °/90 °) and UV light proved to have the most detrimental impact. Changes in the stability of the rAAV/DJ/CAG structure are manifested by variations in the number of genome copies (gc) and GFP+ cells. Temperature fluctuations resulted in differences in the number of gc while maintaining a similar number of GFP+ cells, which may indicate specific changes in the rAAV/DJ/CAG structure, triggering disorders or degradation in the vector entry. This study provides interesting insights into rAAV/DJ/CAG, and the implications of these findings provide a basis for developing new protocols in cancer gene therapy.


Assuntos
Dependovirus , Vetores Genéticos , Dependovirus/genética , Terapia Genética , Vetores Genéticos/genética , Temperatura , Transdução Genética
9.
Bull Cancer ; 108(10S): S162-S167, 2021 Oct.
Artigo em Francês | MEDLINE | ID: mdl-34920799

RESUMO

CAR-T cells belong to a new class of biological medicines, referred to as Advanced Therapy Medicinal Products (ATMPs). Despite the cellular component, according to the regulatory definition, CAR-T cells are gene therapy medicines, a sub-category of ATMPs, since their therapeutic effect is the result of their genetic modification. The specificity and the complexity of these innovative drugs have required a complete reorganization of the hospital and pharmaceutical circuits, from the cell collection to the drug administration to the patient. Indeed, increased interaction and collaboration between different healthcare professionals is essential in order to guarantee the quality and safety of these innovative medicines.


Assuntos
Engenharia Celular/legislação & jurisprudência , Terapia Genética/legislação & jurisprudência , Imunoterapia Adotiva/legislação & jurisprudência , Receptores de Antígenos Quiméricos , Linfócitos T , Composição de Medicamentos/normas , Indústria Farmacêutica/legislação & jurisprudência , Indústria Farmacêutica/normas , Europa (Continente) , França , Terapia Genética/métodos , Humanos , Imunoterapia Adotiva/métodos , Linfócitos T/imunologia , Linfócitos T/transplante
11.
Blood Adv ; 5(23): 5452, 2021 12 14.
Artigo em Inglês | MEDLINE | ID: mdl-34905048

RESUMO

Sickle cell disease (SCD) is an inherited blood condition resulting from abnormal hemoglobin production. It is one of the most common genetic diseases in the world. The clinical manifestations are variable and range from recurrent acute and debilitating painful crises to life-threatening pulmonary, cardiovascular, renal, and neurologic complications. The only curative treatment of SCD at this time is bone marrow transplantation (also called hematopoietic stem cell transplantation) using healthy blood stem cells from an unaffected brother or sister or from an unrelated donor if one can be identified who is a match in tissue typing. Unfortunately, only a minority of patients with sickle cell has such a donor available. The use of autologous hematopoietic stem cells and alternative types of genetic modifications is currently under study in clinical research trials for this disease. The approaches include the use of viral vectors to express globin genes that are modified to prevent sickle hemoglobin polymerization or to express interfering RNAs to "flip the switch" in adult red cells from adult ß-sickle hemoglobin to fetal hemoglobin using a physiologic switch, and several gene editing approaches with the goal of inducing fetal hemoglobin or correcting/modifying the actual sickle mutation. In this audio review, we will discuss these different approaches and review the current progress of curative therapy for SCD using gene therapy.


Assuntos
Anemia Falciforme , Terapia Genética , Anemia Falciforme/genética , Anemia Falciforme/terapia , Hemoglobina Fetal , Hemoglobina Falciforme/genética , Humanos , Doadores não Relacionados
12.
Int J Nanomedicine ; 16: 7697-7709, 2021.
Artigo em Inglês | MEDLINE | ID: mdl-34819728

RESUMO

Introduction: ß-lactamase (LACTB) is a tumor suppressor gene in various tumors including melanoma. However, it remains challenging to efficiently deliver the LACTB gene into melanoma. Recently, we designed a nonviral nanocarrier iRGD/DOTAP/MPEG-PDLLA (iDPP) that could deliver gene targetedly to melanoma efficiently without obvious adverse effects. Methods: In this study, the tumor-targeted nanoparticle iDPP was prepared to deliver LACTB gene to treat melanoma in vitro and in vivo. First, the expression level of LACTB in 6 clinical specimens of melanoma patients was evaluated. Subsequently, the characteristics of iDPP/LACTB nanocomplexes were studied. Afterwards, the in vitro and in vivo anti-tumor efficacy of the iDPP/LACTB nanocomplexes were explored utilizing the B16-F10 mouse melanoma cell line and the B16-F10 subcutaneous melanoma model. Results: Compared with the normal epithelium, the expression level of LACTB in melanoma tissues was significantly downregulated. In vitro B16-F10 cell tests showed iDPP/LACTB nanocomplexes could increase the mRNA levels of P21, Bid, Bax, Pidd1, and Sival genes and up-regulate the p53 signaling pathway of melanoma cells, thus promoting cell apoptosis and blocking the cell cycle. Injected intravenously, iDPP nanoparticles could deliver DNA to the subcutaneous melanoma targetedly. Based on in vivo mouse xenograft model, iDPP/LACTB nanocomplexes could effectively inhibit tumor proliferation and induce tumor apoptosis, thus significantly inhibiting melanoma growth (tumor inhibition rate is about 68%) in the subcutaneous B16-F10 melanoma model. Conclusion: The downregulated LACTB might be a potential target for melanoma therapy. The iDPP/LACTB nanocomplexes could inhibit the growth of the mouse melanoma without obvious side effects, which provide a new option for melanoma gene therapy research.


Assuntos
Antineoplásicos , Melanoma Experimental , Nanopartículas , Animais , Antineoplásicos/farmacologia , Apoptose , Linhagem Celular Tumoral , Terapia Genética , Humanos , Melanoma Experimental/tratamento farmacológico , Proteínas de Membrana/genética , Camundongos , Camundongos Endogâmicos C57BL , Proteínas Mitocondriais/genética , beta-Lactamases/farmacologia , beta-Lactamases/uso terapêutico
13.
Cancer Treat Rev ; 101: 102310, 2021 Dec.
Artigo em Inglês | MEDLINE | ID: mdl-34757307

RESUMO

PURPOSE: Current guidelines recommend somatic genomic sequencing for patients with advanced pancreatic cancer to identify targetable alterations amenable to targeted therapy. The benefit of somatic genomic sequencing in pancreatic cancer remains unclear. This study aims to assess the evidence supporting genomic sequencing to inform treatment selection for patients with advanced pancreatic cancer. METHODS: A systematic review identified prospective studies of exocrine pancreatic cancer patients published before August 2020 which conducted genomic sequencing to inform treatment selection. Outcomes of interest included the proportion of patients with targetable alterations, the proportion that received targeted treatments, and the impact of targeted treatments on overall survival. Meta-analysis for proportions and hazard ratios was performed using Dersimonian and Laird random effect models. RESULTS: 19 studies (representing 2048 pancreatic cancer patients) were included. Sequencing methodologies, definitions of targetable alterations, and approaches treatment selection varied across studies and were incompletely reported. 590 of 1382 sequenced patients harboured a targetable alteration (random effects meta-analysis estimate of the proportion 0.46, 95% confidence interval 0.32-0.61). The proportion of patients with targetable alterations was highly heterogenous between studies (I2 93%, P < 0.001). 91 of 1390 patients received a matched therapy based on their targetable alterations (random effects meta-analysis estimate of the proportion 0.12, 95% CI 0.06-0.23). One observational study reported an overall survival benefit of matched therapy. CONCLUSIONS: Genomic sequencing frequently identifies targetable alterations in pancreatic cancers. Further research is required to standardize the definitions of targetable alterations, the approach to treatment matching, and quantify the benefit of targeted therapy.


Assuntos
Terapia Genética/métodos , Terapia de Alvo Molecular/métodos , Neoplasias Pancreáticas , Análise de Sequência de DNA/métodos , Reparo Gênico Alvo-Dirigido/métodos , Humanos , Estadiamento de Neoplasias , Neoplasias Pancreáticas/genética , Neoplasias Pancreáticas/patologia , Neoplasias Pancreáticas/terapia , Seleção de Pacientes
14.
Blood ; 138(18): 1645-1646, 2021 11 04.
Artigo em Inglês | MEDLINE | ID: mdl-34734998
15.
Rom J Morphol Embryol ; 62(1): 5-11, 2021.
Artigo em Inglês | MEDLINE | ID: mdl-34609404

RESUMO

Fabry disease represents an X-linked inherited disorder resulting in the accumulation of globotriaosylceramide (Gb3). This review explains the clinical manifestations and the possible therapies for this condition. Fabry disease is considered the second most frequent lysosomal storage disease. More than 1000 mutations of the galactosidase alpha (GLA) gene associated with this disorder have been identified. Pain, either episodic crises or chronic pain, is one of the earliest symptoms in Fabry disease. Gastrointestinal, ocular, ear or skeletal manifestations may complete the clinical picture. Cardiac and renal involvements are the most severe complications leading to organ failure and death. The cerebrovascular lesions may result in severe symptoms including stroke at younger ages. The diagnosis of Fabry disease may be put by enzymatic assays of the α-galactosidase A (AGAL-A) activity in plasma or leukocytes but genetic analysis remains the "gold standard" in identifying the precise mutation and even guiding the treatment. Enzyme replacement therapy (ERT) was the first step in treating subjects with Fabry disease. It proved important decrease of the number of sever clinical events and reduction of symptoms. Chemical chaperone therapy has many advantages including oral administration and was already approved in Europe and US, but it is suitable only for subjects with amenable mutations. Gene therapies (either ex vivo or in vivo) promise to represent a new era for many disorders including Fabry disease, the preliminary data being encouraging. Although many steps were taken in understanding the pathogeny of Fabry disease, future research is needed especially in the field of therapeutic approaches.


Assuntos
Doença de Fabry , Terapia de Reposição de Enzimas , Doença de Fabry/diagnóstico , Doença de Fabry/genética , Doença de Fabry/terapia , Terapia Genética , Humanos , Mutação/genética , alfa-Galactosidase/genética , alfa-Galactosidase/uso terapêutico
16.
Nat Commun ; 12(1): 5897, 2021 10 08.
Artigo em Inglês | MEDLINE | ID: mdl-34625552

RESUMO

Adenine base editors (ABE) are genome-editing tools that have been harnessed to introduce precise A•T to G•C conversion. However, the low activity of ABE at certain sites remains a major bottleneck that precludes efficacious applications. Here, to address it, we develop a directional screening system in human cells to evolve the deaminase component of the ABE, and identify three high-activity NG-ABEmax variants: NG-ABEmax-SGK (R101S/D139G/E140K), NG-ABEmax-R (Q154R) and NG-ABEmax-K (N127K). With further engineering, we create a consolidated variant [NG-ABEmax-KR (N127K/Q154R)] which exhibit superior editing activity both in human cells and in mouse disease models, compared to the original NG-ABEmax. We also find that NG-ABEmax-KR efficiently introduce natural mutations in gamma globin gene promoters with more than four-fold increase in editing activity. This work provides a broadly applicable, rapidly deployable platform to directionally screen and evolve user-specified traits in base editors that extend beyond augmented editing activity.


Assuntos
Adenina , Edição de Genes , Animais , Modelos Animais de Doenças , Feminino , Terapia Genética , Células HEK293 , Humanos , Masculino , Camundongos , Camundongos Endogâmicos C57BL , Fenótipo , gama-Globinas/genética
17.
Clin Obstet Gynecol ; 64(4): 850-851, 2021 12 01.
Artigo em Inglês | MEDLINE | ID: mdl-34608016
18.
Nat Med ; 27(10): 1701-1711, 2021 10.
Artigo em Inglês | MEDLINE | ID: mdl-34608334

RESUMO

Spinal muscular atrophy type 1 (SMA1) is a debilitating neurodegenerative disease resulting from survival motor neuron 1 gene (SMN1) deletion/mutation. Onasemnogene abeparvovec (formerly AVXS-101) is a gene therapy that restores SMN production via one-time systemic administration. The present study demonstrates widespread biodistribution of vector genomes and transgenes throughout the central nervous system (CNS) and peripheral organs, after intravenous administration of an AAV9-mediated gene therapy. Two symptomatic infants with SMA1 enrolled in phase III studies received onasemnogene abeparvovec. Both patients died of respiratory complications unrelated to onasemnogene abeparvovec. One patient had improved motor function and the other died shortly after administration before appreciable clinical benefit could be observed. In both patients, onasemnogene abeparvovec DNA and messenger RNA distribution were widespread among peripheral organs and in the CNS. The greatest concentration of vector genomes was detected in the liver, with an increase over that detected in CNS tissues of 300-1,000-fold. SMN protein, which was low in an untreated SMA1 control, was clearly detectable in motor neurons, brain, skeletal muscle and multiple peripheral organs in treated patients. These data support the fact that onasemnogene abeparvovec has effective distribution, transduction and expression throughout the CNS after intravenous administration and restores SMN expression in humans.


Assuntos
Produtos Biológicos/efeitos adversos , Terapia Genética/efeitos adversos , Proteínas Recombinantes de Fusão/efeitos adversos , Atrofias Musculares Espinais da Infância/terapia , Proteína 1 de Sobrevivência do Neurônio Motor/genética , Autopsia , Produtos Biológicos/administração & dosagem , DNA/genética , Feminino , Vetores Genéticos/administração & dosagem , Vetores Genéticos/efeitos adversos , Vetores Genéticos/genética , Humanos , Lactente , Recém-Nascido , Masculino , Neurônios Motores/efeitos dos fármacos , Neurônios Motores/patologia , RNA Mensageiro/genética , Proteínas Recombinantes de Fusão/administração & dosagem , Proteínas Recombinantes de Fusão/genética , Atrofias Musculares Espinais da Infância/genética , Atrofias Musculares Espinais da Infância/mortalidade , Atrofias Musculares Espinais da Infância/patologia , Distribuição Tecidual/efeitos dos fármacos
19.
Clin Obstet Gynecol ; 64(4): 876-885, 2021 12 01.
Artigo em Inglês | MEDLINE | ID: mdl-34618719

RESUMO

Prenatal gene therapy could provide a cure for many monogenic diseases. Prenatal gene therapy has multiple potential advantages over postnatal therapy, including treating before the onset of disease, the ability to induce tolerance and cross the blood-brain barrier. In this chapter, we will describe in utero gene therapy and its rationale, clinical trials of postnatal gene therapy, preclinical studies of in utero gene therapy, and potential risks to the mother and fetus.


Assuntos
Terapias Fetais , Terapia Genética , Feminino , Feto , Humanos , Gravidez
20.
Regen Med ; 16(10): 905-908, 2021 10.
Artigo em Inglês | MEDLINE | ID: mdl-34622685

RESUMO

Geoff MacKay is a pioneer in cell and gene therapy with a track record of successful leadership at innovative biotechs. He is currently the president and CEO of AVROBIO, a clinical-stage lentiviral gene therapy company that treats lysosomal disorders, and a board member of Talaris Therapeutics and Satellos Bioscience. He is also the founding CEO of eGenesis, a biotech that applies CRISPR-Cas9 gene editing to xenotransplantation and the former president and CEO of Organogenesis, a world-leading cell therapy company. Earlier in his career, MacKay spent 11 years at Novartis in senior leadership positions within the global transplantation and immunology franchise.


Assuntos
Terapia Baseada em Transplante de Células e Tecidos , Doenças Raras , Terapia Genética , Humanos , Organogênese , Doenças Raras/genética , Doenças Raras/terapia
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