Source 1review2022Frontiers in Genome Editing
Toolkit/ex vivo stem cell modification and re-transplantation
ex vivo stem cell modification and re-transplantation
Taxonomy: Mechanism Branch / Architecture. Workflows sit above the mechanism and technique branches rather than replacing them.
Summary
Ex vivo stem cell modification and re-transplantation is a clinical delivery workflow in which a patient's own stem cells are isolated, genetically modified outside the body with CRISPR-based approaches, and returned to the same patient. The supplied evidence identifies this format as common among current clinical CRISPR trials.
Usefulness & Problems
Why this is useful
This workflow is useful because it provides a clinically used route for applying CRISPR editing to patient-derived stem cells before re-transplantation. The evidence also indicates that this setting can incorporate sequencing-based genomic surveillance to detect CRISPR-associated genomic effects, including low-frequency large-scale events.
Problem solved
It addresses the delivery and handling problem of how to perform CRISPR modification in a patient's own stem cells within a clinical workflow. The evidence further highlights an associated problem that current workflows inadequately detect large-scale aberrations such as translocations, inversions, deletions, and chromothripsis.
Taxonomy & Function
Primary hierarchy
Mechanism Branch
Architecture: A delivery strategy grouped with the mechanism branch because it determines how a system is instantiated and deployed in context.
Techniques
No technique tags yet.
Target processes
editingImplementation Constraints
The documented workflow consists of ex vivo isolation of a patient's own stem cells, genetic modification, and re-transplantation, consistent with an autologous format. Sequencing-based genomic surveillance is suggested as a practical addition for detecting large-scale CRISPR-associated genomic effects, but the evidence does not specify particular sequencing platforms or assay designs.
Current workflows have difficulty detecting large-scale genomic aberrations, including translocations, inversions, deletions, and chromothripsis. The supplied evidence does not provide quantitative performance data, specific stem cell types, editing reagents, or clinical outcome metrics.
Validation
Cell-freeBacteriaMammalianMouseHumanTherapeuticIndep. Replication
Supporting Sources
Ranked Claims
Large-scale aberrations such as translocations, inversions, deletions, and chromothripsis are more difficult to detect using current workflows, indicating a major unmet need.
However, large-scale aberrations have recently been reported such as translocations, inversions, deletions, and even chromothripsis. These are more difficult to detect using current workflows indicating a major unmet need in the field.
Large-scale aberrations such as translocations, inversions, deletions, and chromothripsis are more difficult to detect using current workflows, indicating a major unmet need.
However, large-scale aberrations have recently been reported such as translocations, inversions, deletions, and even chromothripsis. These are more difficult to detect using current workflows indicating a major unmet need in the field.
Large-scale aberrations such as translocations, inversions, deletions, and chromothripsis are more difficult to detect using current workflows, indicating a major unmet need.
However, large-scale aberrations have recently been reported such as translocations, inversions, deletions, and even chromothripsis. These are more difficult to detect using current workflows indicating a major unmet need in the field.
Large-scale aberrations such as translocations, inversions, deletions, and chromothripsis are more difficult to detect using current workflows, indicating a major unmet need.
However, large-scale aberrations have recently been reported such as translocations, inversions, deletions, and even chromothripsis. These are more difficult to detect using current workflows indicating a major unmet need in the field.
Large-scale aberrations such as translocations, inversions, deletions, and chromothripsis are more difficult to detect using current workflows, indicating a major unmet need.
However, large-scale aberrations have recently been reported such as translocations, inversions, deletions, and even chromothripsis. These are more difficult to detect using current workflows indicating a major unmet need in the field.
Large-scale aberrations such as translocations, inversions, deletions, and chromothripsis are more difficult to detect using current workflows, indicating a major unmet need.
However, large-scale aberrations have recently been reported such as translocations, inversions, deletions, and even chromothripsis. These are more difficult to detect using current workflows indicating a major unmet need in the field.
Large-scale aberrations such as translocations, inversions, deletions, and chromothripsis are more difficult to detect using current workflows, indicating a major unmet need.
However, large-scale aberrations have recently been reported such as translocations, inversions, deletions, and even chromothripsis. These are more difficult to detect using current workflows indicating a major unmet need in the field.
Sequencing-based solutions may be able to detect large-scale CRISPR-associated genomic effects even at low frequencies of occurrence.
we summarize potential sequencing-based solutions that may be able to detect these large-scale effects even at low frequencies of occurrence
Sequencing-based solutions may be able to detect large-scale CRISPR-associated genomic effects even at low frequencies of occurrence.
we summarize potential sequencing-based solutions that may be able to detect these large-scale effects even at low frequencies of occurrence
Sequencing-based solutions may be able to detect large-scale CRISPR-associated genomic effects even at low frequencies of occurrence.
we summarize potential sequencing-based solutions that may be able to detect these large-scale effects even at low frequencies of occurrence
Sequencing-based solutions may be able to detect large-scale CRISPR-associated genomic effects even at low frequencies of occurrence.
we summarize potential sequencing-based solutions that may be able to detect these large-scale effects even at low frequencies of occurrence
Sequencing-based solutions may be able to detect large-scale CRISPR-associated genomic effects even at low frequencies of occurrence.
we summarize potential sequencing-based solutions that may be able to detect these large-scale effects even at low frequencies of occurrence
Sequencing-based solutions may be able to detect large-scale CRISPR-associated genomic effects even at low frequencies of occurrence.
we summarize potential sequencing-based solutions that may be able to detect these large-scale effects even at low frequencies of occurrence
Sequencing-based solutions may be able to detect large-scale CRISPR-associated genomic effects even at low frequencies of occurrence.
we summarize potential sequencing-based solutions that may be able to detect these large-scale effects even at low frequencies of occurrence
Many current clinical CRISPR trials use an ex vivo workflow involving stem cell isolation, modification, and re-transplantation.
many of the current clinical trials using CRISPR involve ex vivo isolation of a patient's own stem cells, modification, and re-transplantation
Many current clinical CRISPR trials use an ex vivo workflow involving stem cell isolation, modification, and re-transplantation.
many of the current clinical trials using CRISPR involve ex vivo isolation of a patient's own stem cells, modification, and re-transplantation
Many current clinical CRISPR trials use an ex vivo workflow involving stem cell isolation, modification, and re-transplantation.
many of the current clinical trials using CRISPR involve ex vivo isolation of a patient's own stem cells, modification, and re-transplantation
Many current clinical CRISPR trials use an ex vivo workflow involving stem cell isolation, modification, and re-transplantation.
many of the current clinical trials using CRISPR involve ex vivo isolation of a patient's own stem cells, modification, and re-transplantation
Many current clinical CRISPR trials use an ex vivo workflow involving stem cell isolation, modification, and re-transplantation.
many of the current clinical trials using CRISPR involve ex vivo isolation of a patient's own stem cells, modification, and re-transplantation
Many current clinical CRISPR trials use an ex vivo workflow involving stem cell isolation, modification, and re-transplantation.
many of the current clinical trials using CRISPR involve ex vivo isolation of a patient's own stem cells, modification, and re-transplantation
Many current clinical CRISPR trials use an ex vivo workflow involving stem cell isolation, modification, and re-transplantation.
many of the current clinical trials using CRISPR involve ex vivo isolation of a patient's own stem cells, modification, and re-transplantation
Approval Evidence
1 source1 linked approval claimfirst-pass slug ex-vivo-stem-cell-modification-and-re-transplantation
many of the current clinical trials using CRISPR involve ex vivo isolation of a patient's own stem cells, modification, and re-transplantation
Source:
workflow contextsupports
Many current clinical CRISPR trials use an ex vivo workflow involving stem cell isolation, modification, and re-transplantation.
many of the current clinical trials using CRISPR involve ex vivo isolation of a patient's own stem cells, modification, and re-transplantation
Source:
Comparisons
Source-backed strengths
The main strength supported by the evidence is that this is already a common workflow in current clinical CRISPR trials, indicating practical clinical relevance. Sequencing-based approaches are also noted as a potential way to detect large-scale CRISPR-associated genomic effects even when they occur at low frequency.
Ranked Citations
- 1.