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Cell & Gene Therapy

Cell & Gene Therapy

The research focuses on the development of advanced therapies, including mesenchymal stem cell therapy, CAR-T cell therapy, gene therapy, and other cell-based approaches for the treatment of genetic diseases, cancer, and chronic conditions.

MSC Therapy

UC-MSC treating Liver Cirrhosis
Cell & Gene Therapy Clinical Phase 1 Gastrointestinal diseases

UC-MSC treating Liver Cirrhosis

Prof. Nguyen Thanh Liem

Umbilical Cord-derived Mesenchymal Stem Cell Infusion in the Management of Adult Liver Cirrhosis
To evaluate the safety and preliminary efficacy of allogeneic umbilical cord-derived MSCs (UC-MSCs) administered via hepatic artery infusion in patients with liver cirrhosis

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Autologous AD-MSC treating Hormone Deficiency in middle ages
Cell & Gene Therapy Clinical Phase 1 Endocrine imbalance

Autologous AD-MSC treating Hormone Deficiency in middle ages

Prof. Nguyen Thanh Liem

Evaluation of Autologous Adipose Derived Mesenchymal Stem Cells (AD-MSCs) Therapy on Sexual Hormone Deficiency in the Middle-aged Patients: an Open-label, Single-group Clinical Trial

MSC treating type II diabetes
Cell & Gene Therapy Clinical Phase 1

MSC treating type II diabetes

Prof. Nguyen Thanh Liem

Open label, single-group clinical trial evaluating the safety and efficacy of mesenchymal stem cell therapy for type 2 diabetes mellitus

Phase I: UC-MSC treatment bronchopulmonary dysplasia
Cell & Gene Therapy Clinical Phase 1 Respiratory diseases

Phase I: UC-MSC treatment bronchopulmonary dysplasia

Prof. Nguyen Thanh Liem & Dr. Trieu Thi Hong Thai

Outcomes of human umbilical cord-derived mesenchymal stem cell infusion in the treatment of bronchopulmonary dysplasia: phase 1 open-label clinical trial 

Autologous BM-MSC therapy combining Psychological education treating Autism
Cell & Gene Therapy Clinical Phase 1

Autologous BM-MSC therapy combining Psychological education treating Autism

Prof. Nguyen Thanh Liem

Autologous Bone Marrow Stem Cell Therapy Combined With Psychological Therapy and Rehabilitation for Autism: An Open Label Controlled Clinical Trial

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Autologous BM-MSC treating cerebral palsy in children
Cell & Gene Therapy Clinical Phase 1 Neurological diseases

Autologous BM-MSC treating cerebral palsy in children

Prof. Nguyen Thanh Liem

Outcomes of autologous Bone Marrow Stem Cell Therapy in the treatment of children with cerebral palsy

Controlled clinical: UC-MSC treating ischemic stroke
Cell & Gene Therapy Clinical Phase 1 Neurological diseases

Controlled clinical: UC-MSC treating ischemic stroke

Prof. Nguyen Thanh Liem & Dr. Phung Nam Lam

Outcomes of umbilical cord blood-derived mesenchymal stem cell infusion in the treatment of neurological sequelae after ischemic stroke: A controlled clinical trial

Phase I/II: Allogenic UC-MSC treating COPD
Cell & Gene Therapy Clinical Phase 1 Respiratory diseases

Phase I/II: Allogenic UC-MSC treating COPD

Ass Prof. Nguyen Hai Anh & Dr. Hoang Minh Duc

Outcomes of safety and efficacy of allogeneic human umbilical cord-derived mesenchymal stem/stromal cells for chronic obstructive pulmonary disease (COPD): a matched case–control, phase I/II trial

Randomized controlled clinical: VICELL 1 treating frailty
Cell & Gene Therapy Clinical Phase 1 Aging

Randomized controlled clinical: VICELL 1 treating frailty

Prof. Nguyen Thanh Liem

Evaluation of the safety and efficacy of umbilical cord-derived mesenchymal stem cell transplantation (VICELL 1) in the treatment of frailty syndrome in the elderly: A randomized controlled clinical trial
To evaluate the effectiveness of intrathecal autologous bone marrow mononuclear cell (BMMNC) therapy combined with education compared with education alone for the treatment of autism spectrum disorder (ASD).

Phase I: AD-MSC (GXIPC1) treating type I diabetes
Cell & Gene Therapy Clinical Phase 1 Autoimmune diseases

Phase I: AD-MSC (GXIPC1) treating type I diabetes

Prof. Nguyen Thanh Liem & Ass.Prof. Nguyen Thi Hoan

Phase I open-label clinical trial to evaluate safety of adipose-derived mesenchymal stem Cell (GXIPC1) transplantation for type 1 diabetes treatment

Phase I/IIa: UC-MSC treating male Sexual Deficiency
Cell & Gene Therapy Clinical Phase 1 Endocrine imbalance

Phase I/IIa: UC-MSC treating male Sexual Deficiency

Dr. Nguyen Thi Tan Sinh & Dr. Hoang Thanh Van

Evaluation of the Safety and Efficacy of Umbilical Cord-Derived Mesenchymal Stem Cell (VCELL 1) for Male Patients With Sexual Deficiency: A Phase I/IIa Randomised Crossover Trial

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Autologous AD-MSC treating Female Sexual Deficiency
Cell & Gene Therapy Clinical Phase 1 Endocrine imbalance

Autologous AD-MSC treating Female Sexual Deficiency

Dr. Nguyen Thi Tan Sinh & Prof. Nguyen Thanh Liem

Evaluation of Autologous Adipose Tissue-derived Mesenchymal Stem Cell Efficacy for the Treatment of Sexual Function Impairment in Female

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CAR-T Therapy

CAR T-Cell Therapy for Leukemia and Lymphoma
Cell & Gene Therapy Clinical Phase 1 Cancer Diagnosis & Therapy

CAR T-Cell Therapy for Leukemia and Lymphoma

PI: Prof. Nguyen Thanh Liem Co-PI: Prof. Nguyen Van Dinh

This Phase I clinical trial aims to evaluate the safety and efficacy of single-dose anti-CD19 CAR T-cell therapy in patients with relapsed or refractory CD19+ non-Hodgkin lymphoma and acute lymphoblastic leukemia

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CAR T-Cell Therapy for Lupus Treatment
Cell & Gene Therapy Clinical Phase 1 Autoimmune diseases

CAR T-Cell Therapy for Lupus Treatment

PI: Prof. Nguyen Thanh Liem Co-PI: Prof. Hoang Thanh Van

This new project aims to develop a potentially curative treatment for lupus, offering patients long-term remission without continuous immunosuppression.

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Gene Therapy

Development of genetic testing for cardiovascular diseases
Cell & Gene Therapy Preclinical Cardiovascular diseases

Development of genetic testing for cardiovascular diseases

Dr. Dao Thi Mai Lan

Identify the genetic variants cause of cardiovascular diseases by whole exome sequencing and developing and benchmarking the whole exome test for clinical diagnosis of inherited cardiovascular diseases.

Development of genetic testing for drug-resistent epilepsy
Cell & Gene Therapy Preclinical Neurological diseases

Development of genetic testing for drug-resistent epilepsy

Dr. Dao Thi Mai Lan

Identify the genetic variations in pediatric patients with drug resistance epilepsy and Developing and benchmarking the whole exome test for clinical diagnosis of drug resistance epilepsy

Edited-CD34+ cells for treatment of Beta thalassemia
Cell & Gene Therapy Clinical Phase 1 Blood diseases

Edited-CD34+ cells for treatment of Beta thalassemia

PI:Dr. Dao Thi Mai Lan Co-PI: Dr. Bach Quoc Khanh

Automated generation of CRISPR-Cas9 gene-edited CD34+ cells for clinical treatment of Beta thalassemia (VINIF.DA139)
To research and apply gene-editing technology in combination with an automated cell manufacturing system to edit hematopoietic stem cells from patients with beta thalassemia, meeting required standards and establishing a foundation for the implementation of clinical trials and the development of gene therapy in Vietnam.

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Other Cell & Gene therapies

Matched case-control: Autologous BM-MSC treating neurological sequelae post TBI
Cell & Gene Therapy Clinical Phase 1 Neurological diseases

Matched case-control: Autologous BM-MSC treating neurological sequelae post TBI

Prof. Nguyen Thanh Liem

Outcomes of safety and efficacy of autologous bone marrow mononuclear cell infusion in the treatment of neurological sequelae after traumatic brain injury: matched case-control clinical trial
To evaluate the safety and efficacy of autologous bone marrow mononuclear cell administration in treating neurological sequelae after traumatic brain injury

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Phase II: Autologous BM-MSC combining Rehabilitation for children with cerebral palsy
Cell & Gene Therapy Clinical Phase 1 Neurological diseases

Phase II: Autologous BM-MSC combining Rehabilitation for children with cerebral palsy

Prof Nguyen Thanh Liem

Autologous Bone Marrow Mononuclear Cell Transplantation Combined With Rehabilitation Intervention for children with cerebral Palsy (CP): A phase 2 Randomised Controlled trial
A randomized clinical trial in 58 patients comparing intrathecal autologous bone marrow mononuclear cell infusion combined with rehabilitation with a control group. Group A receives infusions at baseline and after 6 months, while Group B serves as the control for 9 months and then receives infusions at months 9 and 15, with follow up until 18 months.

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