Las enfermedades degenerativas de la retina (EDR) son un grupo heterogéneo de patologías, algunas hereditarias y otras con un origen complejo, que producen una pérdida progresiva de las células de la retina con la consiguiente pérdida de la agudeza visual pudiendo avanzar hasta producir una ceguera total. En los últimos años y gracias al...
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November 18, 2021 (v1)PublicationUploaded on: December 4, 2022
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April 14, 2023 (v1)Publication
Neural stem cells represent an attractive tool for the development of regenerative therapies and are being tested in clinical trials for several neurological disorders. Human neural stem cells can be isolated from the central nervous system or can be derived in vitro from pluripotent stem cells. Embryonic sources are ethically controversial and...
Uploaded on: April 15, 2023 -
January 16, 2019 (v1)Publication
Retinitis pigmentosa (RP) is an inherited retinal degenerative disease. Mutations in EYS have been associated with autosomal recessive RP. The human iPS cell line, CABi002-A, derived from peripheral blood mononuclear cells from a patient carrying a heterozygous double mutation in EYS gene was generated by non-integrative reprogramming...
Uploaded on: December 4, 2022 -
November 16, 2022 (v1)Publication
Retinal dystrophies associated to mutations in the CRB1 gene comprise a wide array of clinical presentations. A blood sample from a patient with a family history of CRB1-retinal dystrophy was used to prepare the iPSC line ESi082-A. The genotype of the donor, affected of a perifoveal-bilateral macular dystrophy includes one frameshift deletion...
Uploaded on: March 24, 2023 -
November 22, 2022 (v1)Publication
Mutations in the EYS gene are one of the major causes of autosomal recessive retinitis pigmentosa. EYS-retinopathy presents a severe clinical phenotype, and patients currently have no therapeutic options. The progress in personalised medicine and gene and cell therapies hold promise for treating this degenerative disease. However, lack of...
Uploaded on: March 24, 2023 -
June 7, 2023 (v1)Publication
Background: There remains much interest in improving cryopreservation techniques for advanced therapy medicinal products (ATMPs). Recently, human platelet lysate (hPL) has emerged as a promising candidate to replace fetal bovine serum (FBS) as a xeno-free culture supplement for the expansion of human cell therapy products. Whether hPL can also...
Uploaded on: June 9, 2023 -
May 23, 2023 (v1)Publication
Animal models currently used to test the efficacy and safety of cell therapies, mainly murine models, have limitations as molecular, cellular, and physiological mechanisms are often inherently different between species, especially in the brain. Therefore, for clinical translation of cell-based medicinal products, the development of alternative...
Uploaded on: May 26, 2023 -
February 6, 2023 (v1)Publication
Age-related macular degeneration (AMD) is the leading cause of adult blindness in developed countries and is characterized by progressive degeneration of the macula, the central region of the retina. A human induced pluripotent stem cell (hiPSC) line was derived from peripheral blood mononuclear cells (PBMCs) from a patient with a clinical...
Uploaded on: March 1, 2023