Neural Transplantation for the Treatment of Huntington’s Disease

Barker, Roger A.; Swain, Rachel

doi:10.17925/enr.2010.05.02.41

Cited by 4 publications

(4 citation statements)

References 28 publications

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“…Al menos 5 ensayos clínicos implementaron el mismo protocolo de seguimiento (CAPIT-HD), mostrando resultados variables, obteniendo en algunos casos mejoras clínicas sostenidas hasta seis años después del trasplante en comparación con pacientes no trasplantados, mientras que en otros casos no se observaron diferencias significativas en comparación con los controles. 68 El uso de células madre pluripotentes (CPM) como fuente celular sustitutiva podría constituir una alternativa al uso de células de tejido fetal, debido a su capacidad para diferenciarse en las 3 capas germinales primarias y dividirse indefinidamente sin transformarse, lo que ofrece un suministro ilimitado de material de partida. Las células para este propósito se pueden obtener de los propios pacientes con EH, por lo tanto, contendrán la elongación de CAG en el gen Htt, así como la corrección genética previa requerida para generar células con un número normal de repeticiones de CAG a partir de ellas.…”

Section: Síntomas Motoresunclassified

Current Knowledge and Future Directions in Huntington’s Disease

et al. 2022

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Introduction: Huntington's Disease (HD) is a hereditary, neurodegenerative disorder due to abnormal repeats of the CAG triplet in the IT-15 gene. It is characterized by a triad of progressive motor, psychiatric and cognitive symptoms, resulting from striatal neuronal loss. The impact of HD in Latin America is evidenced by the prevalence rates in Maracaibo (Venezuela) and Juan de Acosta (Colombia), which are the highest recorded in the world. This contrasts with the social abandonment and the scarce intervention of local governments. Aim: Provide an approach to the most relevant aspects of HD from its pathogenesis and associated genetic polymorphisms, to the current treatment options. Methodology: A literature review was performed of the state of the art of prognosis and treatment strategies in HD, including the identification of different polymorphic markers in the genes coding for UCHL1, HIP1, PGC1α, GRIK2, TBP, BDNF, among others, and its associations in the evolution of the disease in the presence of abnormal CAG repeats. Conclusion: Even though in the last decades many researchers have focused on the development of curative treatment, there is no effective therapeutic target to prevent the clinical onset of the disease or to delay its progression. Instead, current pharmacological management is palliative, the evidence to generalize the surgical approaches is insufficient. Newly, the fetal neural cell transplantation into the striatum is offered as the surgical option that provides hope for the development of a true disease-modifying treatment.

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Section: Síntomas Motoresunclassified

Current Knowledge and Future Directions in Huntington’s Disease

et al. 2022

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“…The patients were followed up with the CAPIT-HD protocol and compared to non-transplanted HD patients. At 2 years post-transplant they were observed to show no significant improvement (Unpublished data; Barker and Swain, 2010 ).…”

Section: Clinical Studies Of Striatal Allografting In Huntington’s DImentioning

confidence: 99%

The Current Status of Neural Grafting in the Treatment of Huntington?s Disease. A Review

Wijeyekoon¹,

Barker²

2011

Front. Integr. Neurosci.

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Huntington’s disease (HD) is a devastating, fatal, autosomal dominant condition in which the abnormal gene codes for a mutant form of huntingtin that causes widespread neuronal dysfunction and death. This leads to a clinical presentation, typically in midlife, with a combination of motor, psychiatric, cognitive, metabolic, and sleep abnormalities, for which there are some effective symptomatic therapies that can produce some transient benefits. The disease, though, runs a progressive course over a 20-year period ultimately leading to death, and there are currently no proven disease modifying therapies. However whilst the neuronal dysfunction and loss affects much of the central nervous system, the striatum is affected early on in the disease and is one of the areas most affected by the pathogenic process. As a result the prospect of treating HD using neural transplants of striatal tissue has been explored and to date the clinical data is inconclusive. In this review we discuss the rationale for treating HD using this approach, before discussing the clinical trial data and what we have learnt to date using this therapeutic strategy.

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“…Further studies for clinical efficacy are necessary, particularly to verify cell migration. Details of neural brain cell transplantation into HD patients have been reported in 2010 on six separate trials, including the above pilot test [65]. In summary, operatively, from 3 to 7 patients received neural implants into the brain striatum.…”

Section: Clinical Trialsmentioning

confidence: 99%

Extrapolating Stem Cell Potential into Therapy: Current Parameters for Treating Neurological Defects

Tweedell¹

2011

J Bioengineer & Biomedical Sci

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Stem cell use in regenerative medicine has its basis on experimental procedures developed in animals. Several natural stem cells from embryos, fetal and adult tissues and induced pluripotent stem cells have evolved as choices to replace or repair one or more cell types in human maladies. The roles that stem cells serve as precursors is incumbent upon establishing their potential, reliability and efficacy before they are selected as therapeutic agents in humans with specific human diseases. A group of neurodegenerative diseases with related cell deficiencies, multiple sclerosis, amyotrophic lateral sclerosis, Huntington disease and Parkinson disease are reviewed at the current preclinical potential as prospects for stem cell therapy and compared to present clinical trials on human patients with degenerative diseases. Only a limited number of native stem cell sources have been utilized and early results indicate their safety and moderate effectiveness after transplantation. With continued preclinical research, the usefulness of induced pluripotent stem cells will expand the options.

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Neural Transplantation for the Treatment of Huntington’s Disease

Cited by 4 publications

References 28 publications

Current Knowledge and Future Directions in Huntington’s Disease

Current Knowledge and Future Directions in Huntington’s Disease

The Current Status of Neural Grafting in the Treatment of Huntington?s Disease. A Review

Extrapolating Stem Cell Potential into Therapy: Current Parameters for Treating Neurological Defects

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